Informacja

Drogi użytkowniku, aplikacja do prawidłowego działania wymaga obsługi JavaScript. Proszę włącz obsługę JavaScript w Twojej przeglądarce.

Przeglądasz jako GOŚĆ

Wyszukujesz frazę ""Pyrrolidines"" wg kryterium: Temat


Tytuł :
Glecaprevir-pibrentasvir to treat chronic hepatitis C virus infection in Asia: two multicentre, phase 3 studies- a randomised, double-blind study (VOYAGE-1) and an open-label, single-arm study (VOYAGE-2).
Autorzy :
Wei L; Peking University Hepatology Institute, Peking University People's Hospital, Beijing, China; Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China.
Wang G; Peking University First Hospital, Beijing, China.
Alami NN; AbbVie, North Chicago, IL, USA.
Xie W; Center of Liver Diseases, Beijing Di Tan Hospital, Capital Medical University, Beijing, China.
Heo J; Department of Internal Medicine, College of Medicine, Pusan National University and Medical Research Institute, Busan, South Korea.
Xie Q; Department of Infectious Diseases, Rui Jin Hospital Shanghai Jiaotong University School of Medicine, Shanghai, China.
Zhang M; Shenyang Sixth People's Hospital, Shenyang, China.
Kim YJ; Department of Internal Medicine and Liver Research Institute, Seoul National University College of Medicine, Seoul, South Korea.
Lim SG; National University Healthcare System, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
Fredrick LM; AbbVie, North Chicago, IL, USA.
Lu W; AbbVie, North Chicago, IL, USA.
Liu W; AbbVie, North Chicago, IL, USA.
Kalluri HV; AbbVie, North Chicago, IL, USA.
Krishnan P; AbbVie, North Chicago, IL, USA.
Tripathi R; AbbVie, North Chicago, IL, USA.
Mobashery N; AbbVie, North Chicago, IL, USA.
Burroughs M; AbbVie, North Chicago, IL, USA.
Asatryan A; AbbVie, North Chicago, IL, USA.
Jia J; Liver Research Center, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
Hou J; Department of Infectious Diseases and Hepatology Unit, State Key Laboratory of Organ Failure Research, Guangdong Provincial Key Laboratory of Viral Hepatitis Research, Nanfang Hospital, Southern Medical University, Guangzhou, China. Electronic address: .
Pokaż więcej
Źródło :
The lancet. Gastroenterology & hepatology [Lancet Gastroenterol Hepatol] 2020 Sep; Vol. 5 (9), pp. 839-849. Date of Electronic Publication: 2020 Jul 16.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101690683 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2468-1253 (Electronic) NLM ISO Abbreviation: Lancet Gastroenterol Hepatol Subsets: MEDLINE
MeSH Terms :
Benzimidazoles/*therapeutic use
Hepatitis C, Chronic/*drug therapy
Pyrrolidines/*therapeutic use
Quinoxalines/*therapeutic use
Sulfonamides/*therapeutic use
Adult ; Aged ; Antiviral Agents/administration & dosage ; Antiviral Agents/therapeutic use ; Asia/epidemiology ; Benzimidazoles/administration & dosage ; Benzimidazoles/adverse effects ; Case-Control Studies ; Double-Blind Method ; Drug Combinations ; Drug Therapy, Combination ; Female ; Genotype ; Hepacivirus/drug effects ; Hepacivirus/genetics ; Hepatitis C, Chronic/complications ; Hepatitis C, Chronic/epidemiology ; Humans ; Liver Cirrhosis/drug therapy ; Liver Cirrhosis/epidemiology ; Liver Cirrhosis/etiology ; Male ; Middle Aged ; Placebos/administration & dosage ; Prevalence ; Pyrrolidines/administration & dosage ; Pyrrolidines/adverse effects ; Quinoxalines/administration & dosage ; Quinoxalines/adverse effects ; Respiratory Tract Infections/chemically induced ; Respiratory Tract Infections/epidemiology ; Safety ; Sulfonamides/administration & dosage ; Sulfonamides/adverse effects ; Sustained Virologic Response ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Evocalcet in patients with primary hyperparathyroidism: an open-label, single-arm, multicenter, 52-week, dose-titration phase III study.
Autorzy :
Takeuchi Y; Toranomon Hospital Endocrine Center, 2-2-2, Toranomon, Minato-ku, Tokyo, 105-8470, Japan. .; Okinaka Memorial Institute for Medical Research, Tokyo, Japan. .
Nishida Y; Kyowa Kirin Co., Ltd, Tokyo, Japan.
Kondo Y; Kyowa Kirin Co., Ltd, Tokyo, Japan.
Imanishi Y; Department of Metabolism, Endocrinology and Molecular Medicine, Osaka City University Graduate School of Medicine, Osaka, Japan.
Fukumoto S; Fujii Memorial Institute of Medical Sciences, Institute of Advanced Medical Sciences, Tokushima University, Tokushima, Japan.
Pokaż więcej
Źródło :
Journal of bone and mineral metabolism [J Bone Miner Metab] 2020 Sep; Vol. 38 (5), pp. 687-694. Date of Electronic Publication: 2020 Apr 09.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study
Journal Info :
Publisher: Japanese Society of Bone and Mineral Metabolism Country of Publication: Japan NLM ID: 9436705 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1435-5604 (Electronic) Linking ISSN: 09148779 NLM ISO Abbreviation: J. Bone Miner. Metab. Subsets: MEDLINE
MeSH Terms :
Hyperparathyroidism, Primary/*drug therapy
Naphthalenes/*therapeutic use
Pyrrolidines/*therapeutic use
Calcium/blood ; Creatinine/blood ; Dose-Response Relationship, Drug ; Female ; Humans ; Hyperparathyroidism, Primary/blood ; Male ; Middle Aged ; Naphthalenes/administration & dosage ; Naphthalenes/adverse effects ; Naphthalenes/pharmacology ; Parathyroid Hormone/blood ; Phosphates/blood ; Pyrrolidines/administration & dosage ; Pyrrolidines/adverse effects ; Pyrrolidines/pharmacology
Czasopismo naukowe
Tytuł :
Identification of high-affinity inhibitors of SARS-CoV-2 main protease: Towards the development of effective COVID-19 therapy.
Autorzy :
Mohammad T; Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India.
Shamsi A; Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India.
Anwar S; Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India.
Umair M; Department of Computer Science, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India.
Hussain A; Department of Pharmacognosy, College of Pharmacy, King Saud University, Riyadh, 11451, Saudi Arabia.
Rehman MT; Department of Pharmacognosy, College of Pharmacy, King Saud University, Riyadh, 11451, Saudi Arabia.
AlAjmi MF; Department of Pharmacognosy, College of Pharmacy, King Saud University, Riyadh, 11451, Saudi Arabia.
Islam A; Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India.
Hassan MI; Centre for Interdisciplinary Research in Basic Sciences, Jamia Millia Islamia, Jamia Nagar, New Delhi, 110025, India. Electronic address: .
Pokaż więcej
Źródło :
Virus research [Virus Res] 2020 Oct 15; Vol. 288, pp. 198102. Date of Electronic Publication: 2020 Jul 24.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Elsevier Science Country of Publication: Netherlands NLM ID: 8410979 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1872-7492 (Electronic) Linking ISSN: 01681702 NLM ISO Abbreviation: Virus Res Subsets: MEDLINE
MeSH Terms :
Aminoglycosides/*chemistry
Antiviral Agents/*chemistry
Betacoronavirus/*chemistry
Protease Inhibitors/*chemistry
Pyrrolidines/*chemistry
Viral Nonstructural Proteins/*antagonists & inhibitors
Aminoglycosides/metabolism ; Antiviral Agents/metabolism ; Betacoronavirus/enzymology ; Catalytic Domain ; Coronavirus Infections/drug therapy ; Cysteine Endopeptidases/chemistry ; Cysteine Endopeptidases/genetics ; Cysteine Endopeptidases/metabolism ; Drug Discovery ; Gene Expression ; Humans ; Molecular Docking Simulation ; Molecular Dynamics Simulation ; Pandemics ; Pneumonia, Viral/drug therapy ; Protease Inhibitors/metabolism ; Protein Binding ; Protein Conformation, alpha-Helical ; Protein Conformation, beta-Strand ; Protein Interaction Domains and Motifs ; Pyrrolidines/metabolism ; Substrate Specificity ; Thermodynamics ; User-Computer Interface ; Viral Nonstructural Proteins/chemistry ; Viral Nonstructural Proteins/genetics ; Viral Nonstructural Proteins/metabolism
SCR Disease Name :
COVID-19
SCR Organism :
severe acute respiratory syndrome coronavirus 2
Czasopismo naukowe
Tytuł :
Systematic review and network meta-analyses of third-line treatments for metastatic colorectal cancer.
Autorzy :
Walter T; Cancer Research Center of Lyon, University of Lyon, Claude Bernard University, Lyon, France.; Service d'oncologie médicale, Hôpital E. Herriot, Hospices Civils de Lyon, Lyon, France.
Hawkins NS; Institute of Health and Wellbeing, University of Glasgow, Glasgow, UK.
Pollock RF; Covalence Research Ltd, 51 Hayes Grove, London, SE22 8DF, UK. .
Colaone F; Sirtex Medical United Kingdom Ltd, London, UK.
Shergill S; Sirtex Medical United Kingdom Ltd, London, UK.
Ross PJ; Department of Medical Oncology, Guy's and St Thomas' NHS Foundation Trust, London, UK.; Department of Oncology, King's College Hospital NHS Foundation Trust, London, UK.
Pokaż więcej
Źródło :
Journal of cancer research and clinical oncology [J Cancer Res Clin Oncol] 2020 Oct; Vol. 146 (10), pp. 2575-2587. Date of Electronic Publication: 2020 Jul 27.
Typ publikacji :
Journal Article; Meta-Analysis; Systematic Review
Journal Info :
Publisher: Springer-Verlag Country of Publication: Germany NLM ID: 7902060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1432-1335 (Electronic) Linking ISSN: 01715216 NLM ISO Abbreviation: J. Cancer Res. Clin. Oncol. Subsets: MEDLINE
MeSH Terms :
Antineoplastic Combined Chemotherapy Protocols/*therapeutic use
Brachytherapy/*methods
Colorectal Neoplasms/*diet therapy
Colorectal Neoplasms/*radiotherapy
Phenylurea Compounds/*therapeutic use
Pyridines/*therapeutic use
Pyrrolidines/*therapeutic use
Trifluridine/*therapeutic use
Uracil/*analogs & derivatives
Yttrium Radioisotopes/*administration & dosage
Double-Blind Method ; Drug Combinations ; Humans ; Microspheres ; Neoplasm Metastasis ; Network Meta-Analysis ; Palliative Care/methods ; Phenylurea Compounds/administration & dosage ; Progression-Free Survival ; Pyridines/administration & dosage ; Pyrrolidines/administration & dosage ; Randomized Controlled Trials as Topic ; Trifluridine/administration & dosage ; Uracil/administration & dosage ; Uracil/therapeutic use
Czasopismo naukowe
Tytuł :
Comparative Effectiveness of Calcimimetic Agents for Secondary Hyperparathyroidism in Adults: A Systematic Review and Network Meta-analysis.
Autorzy :
Palmer SC; University of Otago Christchurch, Christchurch, New Zealand.
Mavridis D; Department of Primary Education, University of Ioannina, Greece; Paris Descartes University, Paris, France.
Johnson DW; University of Queensland at the Princess Alexandra Hospital, Queensland, Australia.
Tonelli M; Cumming School of Medicine, University of Calgary, Calgary, Canada.
Ruospo M; Department of Emergency and Organ Transplantation, University of Bari, Bari, Italy.
Strippoli GFM; Department of Emergency and Organ Transplantation, University of Bari, Bari, Italy; Sydney School of Public Health, The University of Sydney, NSW, Australia. Electronic address: .
Pokaż więcej
Źródło :
American journal of kidney diseases : the official journal of the National Kidney Foundation [Am J Kidney Dis] 2020 Sep; Vol. 76 (3), pp. 321-330. Date of Electronic Publication: 2020 May 28.
Typ publikacji :
Comparative Study; Journal Article; Meta-Analysis; Research Support, Non-U.S. Gov't; Systematic Review
Journal Info :
Publisher: W.B. Saunders Country of Publication: United States NLM ID: 8110075 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1523-6838 (Electronic) Linking ISSN: 02726386 NLM ISO Abbreviation: Am J Kidney Dis Subsets: MEDLINE
MeSH Terms :
Calcimimetic Agents/*therapeutic use
Cinacalcet/*therapeutic use
Hyperparathyroidism, Secondary/*drug therapy
Naphthalenes/*therapeutic use
Peptides/*therapeutic use
Pyrrolidines/*therapeutic use
Adult ; Calcimimetic Agents/adverse effects ; Calcium/blood ; Cardiovascular Diseases/epidemiology ; Cinacalcet/adverse effects ; Comorbidity ; Female ; Fractures, Bone/epidemiology ; Heart Failure/epidemiology ; Humans ; Hyperparathyroidism, Secondary/blood ; Hyperparathyroidism, Secondary/etiology ; Male ; Mortality ; Naphthalenes/adverse effects ; Nausea/chemically induced ; Parathyroid Hormone/blood ; Peptides/adverse effects ; Pyrrolidines/adverse effects ; Randomized Controlled Trials as Topic/statistics & numerical data ; Renal Dialysis/adverse effects ; Renal Insufficiency, Chronic/complications ; Renal Insufficiency, Chronic/therapy ; Treatment Outcome ; Vomiting/chemically induced
Czasopismo naukowe
Tytuł :
Discovery of M Protease Inhibitors Encoded by SARS-CoV-2.
Autorzy :
Hung HC; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Ke YY; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Huang SY; Research Center for Emerging Viral Infections, Chang Gung University, Taoyuan, Taiwan.
Huang PN; Research Center for Emerging Viral Infections, Chang Gung University, Taoyuan, Taiwan.; Division of Infectious Diseases, Department of Pediatrics, Linkou Chang Gung Memorial Hospital, Taoyuan, Taiwan.
Kung YA; Research Center for Emerging Viral Infections, Chang Gung University, Taoyuan, Taiwan.; Division of Infectious Diseases, Department of Pediatrics, Linkou Chang Gung Memorial Hospital, Taoyuan, Taiwan.
Chang TY; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Yen KJ; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Peng TT; Department of Animal Technology Laboratories, Agricultural Technology Research Institute (ATRI), Miaoli, Taiwan.
Chang SE; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Huang CT; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Tsai YR; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Wu SH; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Lee SJ; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan.
Lin JH; Department of Animal Technology Laboratories, Agricultural Technology Research Institute (ATRI), Miaoli, Taiwan.
Liu BS; National Institute of Infectious Diseases and Vaccinology, National Health Research Institutes, Miaoli, Taiwan.
Sung WC; National Institute of Infectious Diseases and Vaccinology, National Health Research Institutes, Miaoli, Taiwan.
Shih SR; Research Center for Emerging Viral Infections, Chang Gung University, Taoyuan, Taiwan .; Department of Laboratory Medicine, Linkou Chang Gung Memorial Hospital, Taoyuan, Taiwan.
Chen CT; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan .
Hsu JT; Institute of Biotechnology and Pharmaceutical Research, National Health Research Institutes, Miaoli, Taiwan .; Department of Biological Science and Technology, National Chiao Tung University, Hsinchu, Taiwan.
Pokaż więcej
Źródło :
Antimicrobial agents and chemotherapy [Antimicrob Agents Chemother] 2020 Aug 20; Vol. 64 (9). Date of Electronic Publication: 2020 Aug 20 (Print Publication: 2020).
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: American Society for Microbiology Country of Publication: United States NLM ID: 0315061 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 1098-6596 (Electronic) Linking ISSN: 00664804 NLM ISO Abbreviation: Antimicrob. Agents Chemother. Subsets: MEDLINE
MeSH Terms :
Antiviral Agents/*chemistry
Betacoronavirus/*drug effects
Cysteine Endopeptidases/*chemistry
Protease Inhibitors/*chemistry
Pyrrolidines/*chemistry
Viral Nonstructural Proteins/*chemistry
Amino Acid Motifs ; Animals ; Antiviral Agents/pharmacology ; Betacoronavirus/pathogenicity ; Catalytic Domain ; Chlorocebus aethiops ; Cysteine Endopeptidases/genetics ; Cysteine Endopeptidases/metabolism ; Gene Expression ; Molecular Docking Simulation ; Protease Inhibitors/pharmacology ; Protein Binding ; Protein Conformation, alpha-Helical ; Protein Conformation, beta-Strand ; Protein Interaction Domains and Motifs ; Pyrrolidines/pharmacology ; Recombinant Proteins/chemistry ; Recombinant Proteins/genetics ; Recombinant Proteins/metabolism ; Thermodynamics ; Vero Cells ; Viral Nonstructural Proteins/antagonists & inhibitors ; Viral Nonstructural Proteins/genetics ; Viral Nonstructural Proteins/metabolism ; Virus Replication/drug effects
SCR Organism :
severe acute respiratory syndrome coronavirus 2
Czasopismo naukowe
Tytuł :
Boceprevir, GC-376, and calpain inhibitors II, XII inhibit SARS-CoV-2 viral replication by targeting the viral main protease.
Autorzy :
Ma C; Department of Pharmacology and Toxicology, College of Pharmacy, The University of Arizona, Tucson, AZ, 85721, USA.
Sacco MD; Department of Molecular Medicine, Morsani College of Medicine, University of South Florida, Tampa, FL, 33612, USA.
Hurst B; Institute for Antiviral Research, Utah State University, Logan, UT, 84322, USA.; Department of Animal, Dairy and Veterinary Sciences, Utah State University, Logan, UT, 84322, USA.
Townsend JA; Department of Chemistry and Biochemistry, The University of Arizona, Tucson, AZ, 85721, USA.
Hu Y; Department of Pharmacology and Toxicology, College of Pharmacy, The University of Arizona, Tucson, AZ, 85721, USA.
Szeto T; Department of Pharmacology and Toxicology, College of Pharmacy, The University of Arizona, Tucson, AZ, 85721, USA.
Zhang X; Department of Molecular Medicine, Morsani College of Medicine, University of South Florida, Tampa, FL, 33612, USA.
Tarbet B; Institute for Antiviral Research, Utah State University, Logan, UT, 84322, USA.; Department of Animal, Dairy and Veterinary Sciences, Utah State University, Logan, UT, 84322, USA.
Marty MT; Department of Chemistry and Biochemistry, The University of Arizona, Tucson, AZ, 85721, USA.
Chen Y; Department of Molecular Medicine, Morsani College of Medicine, University of South Florida, Tampa, FL, 33612, USA. .
Wang J; Department of Pharmacology and Toxicology, College of Pharmacy, The University of Arizona, Tucson, AZ, 85721, USA. .
Pokaż więcej
Źródło :
Cell research [Cell Res] 2020 Aug; Vol. 30 (8), pp. 678-692. Date of Electronic Publication: 2020 Jun 15.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9425763 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1748-7838 (Electronic) Linking ISSN: 10010602 NLM ISO Abbreviation: Cell Res Subsets: MEDLINE
MeSH Terms :
Antiviral Agents/*pharmacology
Betacoronavirus/*enzymology
Coronavirus Infections/*metabolism
Glycoproteins/*pharmacology
Pneumonia, Viral/*metabolism
Proline/*analogs & derivatives
Pyrrolidines/*pharmacology
Viral Nonstructural Proteins/*antagonists & inhibitors
Virus Replication/*drug effects
A549 Cells ; Animals ; Antiviral Agents/chemistry ; Caco-2 Cells ; Chlorocebus aethiops ; Coronavirus Infections/virology ; Cysteine Endopeptidases/chemistry ; Cysteine Endopeptidases/metabolism ; Drug Discovery/methods ; Humans ; Inhibitory Concentration 50 ; Kinetics ; Pandemics ; Pneumonia, Viral/virology ; Proline/pharmacology ; Protein Conformation ; Pyrrolidines/chemistry ; Vero Cells ; Viral Nonstructural Proteins/chemistry ; Viral Nonstructural Proteins/metabolism
SCR Disease Name :
COVID-19
SCR Organism :
severe acute respiratory syndrome coronavirus 2
Czasopismo naukowe
Tytuł :
Acanthophoraine A, a new pyrrolidine alkaloid from the red alga Acanthophora spicifera .
Autorzy :
Lin JL; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Liang YQ; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Liao XJ; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Yang JT; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Li DC; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Huang YL; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Jiang ZH; Department of Pharmacy, Guangzhou General Hospital of Guangzhou Military Command, Guangzhou, P. R. China.
Xu SH; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Zhao BX; Department of Chemistry, College of Chemistry and Materials Science, Jinan University, Guangzhou, P. R. China.
Pokaż więcej
Źródło :
Natural product research [Nat Prod Res] 2020 Jul; Vol. 34 (14), pp. 2065-2070. Date of Electronic Publication: 2019 Feb 20.
Typ publikacji :
Journal Article
Journal Info :
Publisher: Taylor & Francis Health Sciences Country of Publication: England NLM ID: 101167924 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1478-6427 (Electronic) Linking ISSN: 14786419 NLM ISO Abbreviation: Nat. Prod. Res. Subsets: MEDLINE
MeSH Terms :
Alkaloids/*isolation & purification
Pyrrolidines/*isolation & purification
Rhodophyta/*chemistry
Alkaloids/chemistry ; Anti-Infective Agents/chemistry ; Anti-Infective Agents/isolation & purification ; Anti-Infective Agents/pharmacology ; Molecular Conformation ; Molecular Structure ; Pyrrolidines/chemistry ; Spectrum Analysis
Czasopismo naukowe
Tytuł :
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Autorzy :
Balwani M; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Sardh E; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Ventura P; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Peiró PA; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Rees DC; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Stölzel U; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Bissell DM; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Bonkovsky HL; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Windyga J; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Anderson KE; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Parker C; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Silver SM; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Keel SB; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Wang JD; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Stein PE; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Harper P; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Vassiliou D; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Wang B; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Phillips J; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Ivanova A; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Langendonk JG; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Kauppinen R; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Minder E; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Horie Y; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Penz C; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Chen J; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Liu S; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Ko JJ; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Sweetser MT; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Garg P; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Vaishnaw A; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Kim JB; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Simon AR; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Gouya L; From the Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York (M.B.); Porphyria Center Sweden, Center for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); the Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy (P.V.); Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona (P.A.P.); King's College London, King's College Hospital, London (D.C.R., P.E.S.); Klinikum Chemnitz, Chemnitz, Germany (U.S.); the Liver Center and Porphyria Center, University of California, San Francisco, San Francisco (D.M.B., B.W.); the Section on Gastroenterology and Hepatology, Wake Forest University-North Carolina Baptist Medical Center, Winston-Salem (H.L.B.); the Department of Hemostatic Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the University of Texas Medical Branch, Galveston (K.E.A.); the University of Utah, Salt Lake City (C. Parker, J.P.); the University of Michigan, Ann Arbor (S.M.S.); the Department of Medicine, Division of Hematology, University of Washington, Seattle (S.B.K.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); St. Ivan Rilski University Hospital, Sofia, Bulgaria (A.I.); Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, Department of Internal Medicine, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands (J.G.L.); the Department of Medicine, University Hospital of Helsinki, Helsinki, Finland (R.K.); Stadtspital Triemli, Zentrallabor, Zurich, Switzerland (E.M.); Tottori University School of Medicine, Tottori, Japan (Y.H.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, J.C., S.L., J.J.K., M.T.S., P.G., A.V., J.B.K., A.R.S.); and the University of Paris and the Laboratory of Excellence GR-Ex, Paris, and Centre de Référence Maladies Rares Porphyries, Assistance Publique-Hôpitaux de Paris, Colombes - all in France (L.G.).
Pokaż więcej
Corporate Authors :
ENVISION Investigators
Źródło :
The New England journal of medicine [N Engl J Med] 2020 Jun 11; Vol. 382 (24), pp. 2289-2301.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N. Engl. J. Med. Subsets: Core Clinical (AIM); MEDLINE
MeSH Terms :
RNAi Therapeutics*
Acetylgalactosamine/*analogs & derivatives
Aminolevulinic Acid/*urine
Porphobilinogen/*urine
Porphyria, Acute Intermittent/*drug therapy
Pyrrolidines/*therapeutic use
Acetylgalactosamine/adverse effects ; Acetylgalactosamine/therapeutic use ; Adult ; Double-Blind Method ; Fatigue/etiology ; Female ; Humans ; Injections, Subcutaneous ; Least-Squares Analysis ; Liver/drug effects ; Male ; Nausea/etiology ; Pain/etiology ; Patient Outcome Assessment ; Porphyria, Acute Intermittent/complications ; Porphyria, Acute Intermittent/urine ; Pyrrolidines/adverse effects ; Renal Insufficiency, Chronic/chemically induced ; Transaminases/blood
Czasopismo naukowe
Tytuł :
Daridorexant, a new dual orexin receptor antagonist, in elderly subjects with insomnia disorder.
Autorzy :
Zammit G; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany. .
Dauvilliers Y; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany. .
Pain S; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany.
Sebök Kinter D; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany.
Mansour Y; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany.
Kunz D; From the Clinilabs Drug Development Corporation (G.Z.); Icahn School of Medicine at Mount Sinai (G.Z.), New York, NY; Unité du Sommeil (Y.D.), Département de Neurologie, Hôpital Gui-de-Chauliac, Université Montpellier, INSERM 1061, Montpellier, France; Idorsia Pharmaceuticals Ltd (S.P., D.S.K., Y.M.), Allschwil, Switzerland; and Intellux Berlin GmbH (D.K.), Germany.
Pokaż więcej
Źródło :
Neurology [Neurology] 2020 May 26; Vol. 94 (21), pp. e2222-e2232. Date of Electronic Publication: 2020 Apr 27.
Typ publikacji :
Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: Core Clinical (AIM); MEDLINE
MeSH Terms :
Benzimidazoles/*therapeutic use
Pyrrolidines/*therapeutic use
Sleep Initiation and Maintenance Disorders/*drug therapy
Triazoles/*therapeutic use
Aged ; Aged, 80 and over ; Benzimidazoles/adverse effects ; Dose-Response Relationship, Drug ; Double-Blind Method ; Female ; Humans ; Male ; Orexin Receptor Antagonists/therapeutic use ; Polysomnography ; Pyrrolidines/adverse effects ; Treatment Outcome ; Triazoles/adverse effects
Czasopismo naukowe
Tytuł :
Daridorexant: A new medication for insomnia in older adults?
Autorzy :
Molano JRV
Pokaż więcej
Źródło :
Neurology [Neurology] 2020 May 26; Vol. 94 (21), pp. e2287-e2289.
Typ publikacji :
Journal Article
Journal Info :
Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: Core Clinical (AIM); MEDLINE
MeSH Terms :
Benzimidazoles/*therapeutic use
Pyrrolidines/*therapeutic use
Sleep Initiation and Maintenance Disorders/*drug therapy
Triazoles/*therapeutic use
Aged ; Benzimidazoles/adverse effects ; Female ; Humans ; Male ; Patient Education as Topic ; Pyrrolidines/adverse effects ; Triazoles/adverse effects
Czasopismo naukowe
Tytuł :
A positron emission tomography imaging study to confirm target engagement in the lungs of patients with idiopathic pulmonary fibrosis following a single dose of a novel inhaled αvβ6 integrin inhibitor.
Autorzy :
Maher TM; Royal Brompton Hospital, London, UK.; National Heart and Lung Institute, Imperial College London, London, UK.
Simpson JK; GlaxoSmithKline Research and Development, Stevenage, UK.
Porter JC; University College London Hospital, London, UK.
Wilson FJ; GlaxoSmithKline Research and Development, Stevenage, UK.
Chan R; GlaxoSmithKline Research and Development, Stevenage, UK.
Eames R; GlaxoSmithKline Research and Development, Stevenage, UK.
Cui Y; GlaxoSmithKline Research and Development, Stevenage, UK.
Siederer S; GlaxoSmithKline Research and Development, Stevenage, UK.
Parry S; GlaxoSmithKline Research and Development, Stevenage, UK.
Kenny J; GlaxoSmithKline Research and Development, Stevenage, UK.
Slack RJ; GlaxoSmithKline Research and Development, Stevenage, UK.
Sahota J; University College London Hospital, London, UK.
Paul L; Royal Brompton Hospital, London, UK.
Saunders P; Royal Brompton Hospital, London, UK.; National Heart and Lung Institute, Imperial College London, London, UK.
Molyneaux PL; Royal Brompton Hospital, London, UK.; National Heart and Lung Institute, Imperial College London, London, UK.
Lukey PT; GlaxoSmithKline Research and Development, Stevenage, UK.
Rizzo G; Invicro, A Konica Minolta Company, London, UK.
Searle GE; Invicro, A Konica Minolta Company, London, UK.
Marshall RP; GlaxoSmithKline Research and Development, Stevenage, UK.
Saleem A; Invicro, A Konica Minolta Company, London, UK.
Kang'ombe AR; GlaxoSmithKline Research and Development, Stevenage, UK.
Fairman D; GlaxoSmithKline Research and Development, Stevenage, UK.
Fahy WA; GlaxoSmithKline Research and Development, Stevenage, UK.
Vahdati-Bolouri M; GlaxoSmithKline Research and Development, Stevenage, UK. .
Pokaż więcej
Źródło :
Respiratory research [Respir Res] 2020 Mar 26; Vol. 21 (1), pp. 75. Date of Electronic Publication: 2020 Mar 26.
Typ publikacji :
Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial
Journal Info :
Publisher: BioMed Central Ltd Country of Publication: England NLM ID: 101090633 Publication Model: Electronic Cited Medium: Internet ISSN: 1465-993X (Electronic) Linking ISSN: 14659921 NLM ISO Abbreviation: Respir. Res. Subsets: MEDLINE
MeSH Terms :
Butyrates/*therapeutic use
Idiopathic Pulmonary Fibrosis/*drug therapy
Integrins/*antagonists & inhibitors
Naphthyridines/*therapeutic use
Pyrazoles/*therapeutic use
Pyrrolidines/*therapeutic use
Tidal Volume/*drug effects
Administration, Inhalation ; Aged ; Antigens, Neoplasm ; Bayes Theorem ; Butyrates/administration & dosage ; Butyrates/pharmacokinetics ; Double-Blind Method ; Endpoint Determination ; Female ; Humans ; Idiopathic Pulmonary Fibrosis/diagnostic imaging ; Male ; Naphthyridines/administration & dosage ; Naphthyridines/pharmacokinetics ; Nebulizers and Vaporizers ; Positron-Emission Tomography ; Pyrazoles/administration & dosage ; Pyrazoles/pharmacokinetics ; Pyrrolidines/administration & dosage ; Pyrrolidines/pharmacokinetics ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Daridorexant, a New Dual Orexin Receptor Antagonist to Treat Insomnia Disorder.
Autorzy :
Dauvilliers Y; Sleep Unit, Department of Neurology, Neuropsychiatry: Epidemiological and Clinical Research, University of Montpellier, National Institute of Health and Medical Research, Montpellier University Hospital Center, Montpellier, France.
Zammit G; Clinilabs Drug Development Corporation and Icahn School of Medicine at Mount Sinai, New York, NY.
Fietze I; Center of Interdisciplinary Sleep Medicine, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Mayleben D; CTI Clinical Research Center, Cincinnati, OH.
Seboek Kinter D; Clinical Development, Idorsia Pharmaceuticals, Allschwil, Switzerland.
Pain S; Clinical Development, Idorsia Pharmaceuticals, Allschwil, Switzerland.
Hedner J; Sahlgrenska University Hospital, University of Gothenburg, Gothenburg, Sweden.
Pokaż więcej
Źródło :
Annals of neurology [Ann Neurol] 2020 Mar; Vol. 87 (3), pp. 347-356. Date of Electronic Publication: 2020 Feb 05.
Typ publikacji :
Clinical Trial, Phase II; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann. Neurol. Subsets: MEDLINE
MeSH Terms :
Benzimidazoles/*administration & dosage
Pyrrolidines/*administration & dosage
Sleep Initiation and Maintenance Disorders/*drug therapy
Triazoles/*administration & dosage
Adult ; Benzimidazoles/adverse effects ; Benzimidazoles/therapeutic use ; Dose-Response Relationship, Drug ; Double-Blind Method ; Humans ; Middle Aged ; Orexin Receptor Antagonists/adverse effects ; Orexin Receptor Antagonists/therapeutic use ; Pyrrolidines/adverse effects ; Pyrrolidines/therapeutic use ; Treatment Outcome ; Triazoles/adverse effects ; Triazoles/therapeutic use ; Zolpidem/therapeutic use
Czasopismo naukowe
Tytuł :
Antiproliferative activity of diarylnaphthylpyrrolidine derivative via dual target inhibition.
Autorzy :
Verma AK; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India.
Fatima K; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Dudi RK; CSIR-Indian Institute of Integrative Medicine (CSIR-IIIM), Canal Road, Jammu, 180001, India.
Tabassum M; CSIR-Indian Institute of Integrative Medicine (CSIR-IIIM), Canal Road, Jammu, 180001, India.
Iqbal H; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India.
Kumar Y; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Luqman S; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Mondhe DM; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India; CSIR-Indian Institute of Integrative Medicine (CSIR-IIIM), Canal Road, Jammu, 180001, India.
Chanda D; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Khan F; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Shanker K; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India.
Negi AS; CSIR-Central Institute of Medicinal and Aromatic Plants (CSIR-CIMAP), P.O. CIMAP, Kukrail Picnic Spot Road, Lucknow, 226 015, Uttar Pradesh, India; Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, Uttar Pradesh, India. Electronic address: .
Pokaż więcej
Źródło :
European journal of medicinal chemistry [Eur J Med Chem] 2020 Feb 15; Vol. 188, pp. 111986. Date of Electronic Publication: 2019 Dec 20.
Typ publikacji :
Journal Article
Journal Info :
Publisher: Editions Scientifiques Elsevier Country of Publication: France NLM ID: 0420510 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1768-3254 (Electronic) Linking ISSN: 02235234 NLM ISO Abbreviation: Eur J Med Chem Subsets: MEDLINE
MeSH Terms :
Antineoplastic Agents/*pharmacology
Naphthalenes/*pharmacology
Pyrrolidines/*pharmacology
Topoisomerase II Inhibitors/*pharmacology
Tubulin Modulators/*pharmacology
Animals ; Antineoplastic Agents/chemical synthesis ; Antineoplastic Agents/toxicity ; Apoptosis/drug effects ; Breast Neoplasms/drug therapy ; Cell Line, Tumor ; Drug Screening Assays, Antitumor ; Female ; G2 Phase Cell Cycle Checkpoints/drug effects ; Humans ; Male ; Mice ; Molecular Docking Simulation ; Naphthalenes/chemical synthesis ; Naphthalenes/toxicity ; Pyrrolidines/chemical synthesis ; Pyrrolidines/toxicity ; Topoisomerase II Inhibitors/chemical synthesis ; Topoisomerase II Inhibitors/toxicity ; Tubulin Modulators/chemical synthesis ; Tubulin Modulators/toxicity
Czasopismo naukowe
Tytuł :
Current and future role of fedratinib in the treatment of myelofibrosis.
Autorzy :
Ragheb M; Department of Haematology, Guy's & St. Thomas' NHS Foundation Trust, London, UK.
Harrison CN; Department of Haematology, Guy's & St. Thomas' NHS Foundation Trust, London, UK.
McLornan DP; Department of Haematology, Guy's & St. Thomas' NHS Foundation Trust, London, UK.
Pokaż więcej
Źródło :
Future oncology (London, England) [Future Oncol] 2020 Feb; Vol. 16 (6), pp. 175-186. Date of Electronic Publication: 2020 Jan 23.
Typ publikacji :
Journal Article; Review
Journal Info :
Publisher: Future Medicine Ltd Country of Publication: England NLM ID: 101256629 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-8301 (Electronic) Linking ISSN: 14796694 NLM ISO Abbreviation: Future Oncol Subsets: MEDLINE
MeSH Terms :
Janus Kinase 2/*antagonists & inhibitors
Primary Myelofibrosis/*drug therapy
Pyrrolidines/*therapeutic use
Sulfonamides/*therapeutic use
Clinical Trials as Topic ; Drug Development ; Humans ; Pyrazoles/adverse effects ; Pyrazoles/therapeutic use ; Pyrrolidines/adverse effects ; Pyrrolidines/pharmacology ; Sulfonamides/adverse effects ; Sulfonamides/pharmacology ; Treatment Outcome ; Wernicke Encephalopathy/chemically induced
Czasopismo naukowe
Tytuł :
1-Benzylpyrrolidine-3-amine-based BuChE inhibitors with anti-aggregating, antioxidant and metal-chelating properties as multifunctional agents against Alzheimer's disease.
Autorzy :
Wichur T; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Więckowska A; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Więckowski K; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Godyń J; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Jończyk J; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Valdivieso ÁDR; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Panek D; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Pasieka A; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland.
Sabaté R; Department of Pharmacy and Pharmaceutical Technology and Physical Chemistry, Faculty of Pharmacy and Food Science, University of Barcelona, Av Joan XXIII 27-31, 08028, Barcelona, Spain; Institute of Nanoscience and Nanotechnology (IN2UB), University of Barcelona, Av Joan XXIII, S/N, 08028, Barcelona, Spain.
Knez D; University of Ljubljana, Faculty of Pharmacy, Aškerčeva 7, 1000, Ljubljana, Slovenia.
Gobec S; University of Ljubljana, Faculty of Pharmacy, Aškerčeva 7, 1000, Ljubljana, Slovenia.
Malawska B; Faculty of Pharmacy, Jagiellonian University Medical College, Medyczna 9, 30-688, Kraków, Poland. Electronic address: .
Pokaż więcej
Źródło :
European journal of medicinal chemistry [Eur J Med Chem] 2020 Feb 01; Vol. 187, pp. 111916. Date of Electronic Publication: 2019 Nov 26.
Typ publikacji :
Journal Article
Journal Info :
Publisher: Editions Scientifiques Elsevier Country of Publication: France NLM ID: 0420510 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1768-3254 (Electronic) Linking ISSN: 02235234 NLM ISO Abbreviation: Eur J Med Chem Subsets: MEDLINE
MeSH Terms :
Alzheimer Disease/*drug therapy
Amyloid beta-Peptides/*antagonists & inhibitors
Antioxidants/*pharmacology
Butyrylcholinesterase/*metabolism
Chelating Agents/*pharmacology
Cholinesterase Inhibitors/*pharmacology
Pyrrolidines/*pharmacology
Acetylcholinesterase/metabolism ; Alzheimer Disease/metabolism ; Amyloid beta-Peptides/metabolism ; Animals ; Antioxidants/chemical synthesis ; Antioxidants/chemistry ; Benzothiazoles/antagonists & inhibitors ; Chelating Agents/chemical synthesis ; Chelating Agents/chemistry ; Cholinesterase Inhibitors/chemical synthesis ; Cholinesterase Inhibitors/chemistry ; Dose-Response Relationship, Drug ; Electrophorus ; Fluorescence Recovery After Photobleaching ; Horses ; Humans ; Molecular Structure ; Protein Aggregates/drug effects ; Pyrrolidines/chemical synthesis ; Pyrrolidines/chemistry ; Structure-Activity Relationship ; Sulfonic Acids/antagonists & inhibitors
Czasopismo naukowe
Tytuł :
Pharmacokinetics, Safety, and Efficacy of Glecaprevir/Pibrentasvir in Adolescents With Chronic Hepatitis C Virus: Part 1 of the DORA Study.
Autorzy :
Jonas MM; Division of Gastroenterology, Hepatology, and Nutrition, Boston Children's Hospital, Boston, MA.; Department of Pediatrics, Harvard Medical School, Boston, MA.
Squires RH; Division of Gastroenterology, Hepatology, and Nutrition, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA.; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA.
Rhee SM; AbbVie Inc., North Chicago, IL.
Lin CW; Amgen Inc., Thousand Oaks, CA.
Bessho K; Osaka University Hospital, Osaka, Japan.
Feiterna-Sperling C; Charité - Universitaetsmedizin Berlin, Berlin, Germany.
Hierro L; Hospital Universitario La Paz, Madrid, Spain.
Kelly D; Birmingham Women's & Children's Hospital, Birmingham, United Kingdom.
Ling SC; Division of Gastroenterology, Hepatology & Nutrition, The Hospital for Sick Children, and Department of Paediatrics, University of Toronto, Toronto, Ontario, Canada.
Strokova T; Federal Research Centre of Nutrition and Biotechnology, Moscow, Russian Federation.
Del Valle-Segarra A; San Jorge Children's Hospital, San Juan, Puerto Rico.
Lovell S; AbbVie Inc., North Chicago, IL.
Liu W; AbbVie Inc., North Chicago, IL.
Ng TI; AbbVie Inc., North Chicago, IL.
Porcalla A; AbbVie Inc., North Chicago, IL.
Gonzalez YS; AbbVie Inc., North Chicago, IL.
Burroughs M; AbbVie Inc., North Chicago, IL.
Sokal E; Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels, Belgium.
Pokaż więcej
Źródło :
Hepatology (Baltimore, Md.) [Hepatology] 2020 Feb; Vol. 71 (2), pp. 456-462. Date of Electronic Publication: 2019 Aug 13.
Typ publikacji :
Clinical Trial, Phase II; Clinical Trial, Phase III; Journal Article; Research Support, Non-U.S. Gov't
Journal Info :
Publisher: Wiley Country of Publication: United States NLM ID: 8302946 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1527-3350 (Electronic) Linking ISSN: 02709139 Subsets: MEDLINE
MeSH Terms :
Benzimidazoles/*pharmacokinetics
Benzimidazoles/*therapeutic use
Hepatitis C, Chronic/*drug therapy
Pyrrolidines/*pharmacokinetics
Pyrrolidines/*therapeutic use
Quinoxalines/*pharmacokinetics
Quinoxalines/*therapeutic use
Sulfonamides/*pharmacokinetics
Sulfonamides/*therapeutic use
Adolescent ; Benzimidazoles/adverse effects ; Child ; Drug Combinations ; Female ; Humans ; Male ; Pyrrolidines/adverse effects ; Quinoxalines/adverse effects ; Sulfonamides/adverse effects ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Investigation of the metabolism of the selective androgen receptor modulator LGD-4033 in equine urine, plasma and hair following oral administration.
Autorzy :
Cutler C; LGC, Fordham, Cambridgeshire, UK.
Viljanto M; LGC, Fordham, Cambridgeshire, UK.
Hincks P; LGC, Fordham, Cambridgeshire, UK.
Habershon-Butcher J; British Horseracing Authority, London, UK.
Muir T; British Horseracing Authority, London, UK.
Biddle S; LGC, Fordham, Cambridgeshire, UK.
Pokaż więcej
Źródło :
Drug testing and analysis [Drug Test Anal] 2020 Feb; Vol. 12 (2), pp. 247-260. Date of Electronic Publication: 2020 Jan 05.
Typ publikacji :
Journal Article
Journal Info :
Publisher: John Wiley & Sons Country of Publication: England NLM ID: 101483449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1942-7611 (Electronic) Linking ISSN: 19427603 NLM ISO Abbreviation: Drug Test Anal Subsets: MEDLINE
MeSH Terms :
Horses/*metabolism
Nitriles/*metabolism
Performance-Enhancing Substances/*metabolism
Pyrrolidines/*metabolism
Administration, Oral ; Animal Fur/chemistry ; Animal Fur/metabolism ; Animals ; Doping in Sports ; Horses/blood ; Horses/urine ; Nitriles/administration & dosage ; Nitriles/blood ; Nitriles/urine ; Performance-Enhancing Substances/administration & dosage ; Performance-Enhancing Substances/blood ; Performance-Enhancing Substances/urine ; Pyrrolidines/administration & dosage ; Pyrrolidines/blood ; Pyrrolidines/urine ; Receptors, Androgen/metabolism ; Substance Abuse Detection/methods ; Tandem Mass Spectrometry/methods
Czasopismo naukowe
Tytuł :
Investigating the potential use of an ionic liquid (1-Butyl-1-methylpyrrolidinium bis(trifluoromethylsulfonyl)imide) as an anti-fungal treatment against the amphibian chytrid fungus, Batrachochytrium dendrobatidis.
Autorzy :
DiRenzo GV; Department of Ecology, Evolution, & Marine Biology, University of California, Santa Barbara, CA, United States of America.
Chen R; Center for Bioengineering, University of California, Santa Barbara, CA, United States of America.
Ibsen K; Center for Bioengineering, University of California, Santa Barbara, CA, United States of America.; Department of Chemical Engineering, University of California, Santa Barbara, CA, United States of America.; School of Engineering and Applied Sciences, Harvard University Cambridge, Cambridge, MA, United States of America.; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, United States of America.
Toothman M; Department of Ecology, Evolution, & Marine Biology, University of California, Santa Barbara, CA, United States of America.
Miller AJ; Department of Ecology, Evolution, & Marine Biology, University of California, Santa Barbara, CA, United States of America.
Gershman A; Department of Ecology, Evolution, & Marine Biology, University of California, Santa Barbara, CA, United States of America.
Mitragotri S; Center for Bioengineering, University of California, Santa Barbara, CA, United States of America.; Department of Chemical Engineering, University of California, Santa Barbara, CA, United States of America.; School of Engineering and Applied Sciences, Harvard University Cambridge, Cambridge, MA, United States of America.; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, United States of America.
Briggs CJ; Department of Ecology, Evolution, & Marine Biology, University of California, Santa Barbara, CA, United States of America.
Pokaż więcej
Źródło :
PloS one [PLoS One] 2020 Apr 17; Vol. 15 (4), pp. e0231811. Date of Electronic Publication: 2020 Apr 17 (Print Publication: 2020).
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
Journal Info :
Publisher: Public Library of Science Country of Publication: United States NLM ID: 101285081 Publication Model: eCollection Cited Medium: Internet ISSN: 1932-6203 (Electronic) Linking ISSN: 19326203 NLM ISO Abbreviation: PLoS ONE Subsets: MEDLINE
MeSH Terms :
Antifungal Agents/*pharmacology
Anura/*microbiology
Chytridiomycota/*drug effects
Imides/*pharmacology
Ionic Liquids/*pharmacology
Pyrrolidines/*pharmacology
Animals ; Antifungal Agents/therapeutic use ; Cell Survival/drug effects ; Imides/therapeutic use ; Ionic Liquids/therapeutic use ; Mycoses/drug therapy ; Mycoses/microbiology ; Pyrrolidines/therapeutic use ; Skin/microbiology ; Spores, Fungal/drug effects
Czasopismo naukowe
Tytuł :
Trifluridine/tipiracil: A practical guide to its use in the management of refractory metastatic colorectal cancer in Australia.
Autorzy :
Price T; The Queen Elizabeth Hospital, Woodville, SA, Australia.
Burge M; Royal Brisbane Hospital, University of Queensland, QLD, Australia.
Chantrill L; Illawarra Shoalhaven Local Health District, Illawarra Cancer Care Centre, Wollongong Hospital, NSW, Australia.; University of New South Wales Sydney, NSW, Australia.; The Garvan Institute of Medical Research, Sydney, NSW, Australia.
Gibbs P; Walter and Eliza Hall Institute, Parkville, VIC, Australia.; University of Melbourne, Parkville, VIC, Australia.; Western Hospital, Footscray, VIC, Australia.
Pavlakis N; Royal North Shore Hospital, Sydney, NSW, Australia.
Shapiro J; Cabrini Hospital, Melbourne, VIC, Australia.; Monash University, Melbourne, VIC, Australia.
Sjoquist K; NHMRC Clinical Trials Centre, University of Sydney, NSW, Australia.; Cancer Care Centre, St George Hospital, Sydney, NSW, Australia.
Pokaż więcej
Źródło :
Asia-Pacific journal of clinical oncology [Asia Pac J Clin Oncol] 2020 Apr; Vol. 16 Suppl 1, pp. 3-12.
Typ publikacji :
Journal Article
Journal Info :
Publisher: Blackwell Pub. Asia Country of Publication: Australia NLM ID: 101241430 Publication Model: Print Cited Medium: Internet ISSN: 1743-7563 (Electronic) Linking ISSN: 17437555 NLM ISO Abbreviation: Asia Pac J Clin Oncol Subsets: MEDLINE
MeSH Terms :
Antineoplastic Combined Chemotherapy Protocols/*therapeutic use
Colorectal Neoplasms/*drug therapy
Pyrrolidines/*therapeutic use
Quality of Life/*psychology
Trifluridine/*therapeutic use
Uracil/*analogs & derivatives
Antineoplastic Combined Chemotherapy Protocols/pharmacology ; Australia ; Colorectal Neoplasms/pathology ; Drug Combinations ; Female ; Humans ; Male ; Middle Aged ; Prognosis ; Pyrrolidines/pharmacology ; Trifluridine/pharmacology ; Uracil/pharmacology ; Uracil/therapeutic use
Czasopismo naukowe

Ta witryna wykorzystuje pliki cookies do przechowywania informacji na Twoim komputerze. Pliki cookies stosujemy w celu świadczenia usług na najwyższym poziomie, w tym w sposób dostosowany do indywidualnych potrzeb. Korzystanie z witryny bez zmiany ustawień dotyczących cookies oznacza, że będą one zamieszczane w Twoim komputerze. W każdym momencie możesz dokonać zmiany ustawień dotyczących cookies