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Tytuł:
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Autorzy:
Adams D; Neurology Department, CHU Bicêtre, APHP, Université Paris-Saclay, Le Kremlin Bicêtre Cedex, France.
Tournev IL; Department of Neurology, Clinic of Nervous Diseases, University Hospital Aleksandrovska, Medical University, Sofia, Bulgaria.; Department of Cognitive Sciences, New Bulgarian University, Sofia, Bulgaria.
Taylor MS; Department of Clinical Immunology and Allergy, Westmead Hospital and Westmead Clinical School, University of Sydney, Sydney, NSW, Australia.
Coelho T; Hospital de Santo António, Centro Hospitalar Universitário do Porto, Porto, Portugal.
Planté-Bordeneuve V; Neurology - Amyloid Network, CHU Henri Mondor, APHP, University Paris Est - Créteil, Créteil, France.
Berk JL; Boston Medical Center, Boston University, Boston, Massachusetts, USA.
González-Duarte A; Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, México, D.F., México.
Gillmore JD; National Amyloidosis Centre, University College London, Royal Free Hospital, London, UK.
Low SC; Department of Medicine, Division of Neurology, University Malaya Medical Centre, Kuala Lumpur, Malaysia.
Sekijima Y; Department of Medicine (Neurology & Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan.
Obici L; Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy.
Chen C; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Badri P; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Arum SM; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Vest J; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Polydefkis M; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
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Corporate Authors:
HELIOS-A Collaborators
Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2023 Mar; Vol. 30 (1), pp. 1-9. Date of Electronic Publication: 2022 Jul 23.
Typ publikacji:
Randomized Controlled Trial; Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial*/drug therapy
Amyloid Neuropathies, Familial*/genetics
Amyloid Neuropathies, Familial*/complications
Polyneuropathies*/drug therapy
Polyneuropathies*/genetics
Polyneuropathies*/complications
Humans ; Quality of Life ; Prealbumin/genetics
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial.
Autorzy:
Quan D; Department of Neurology, University of Colorado Anschutz, Aurora, CO, USA.
Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Berk JL; Amyloidosis Center, Boston Medical Center, Boston, MA, USA.
Ando Y; Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan.
Aldinc E; Alnylam Pharmaceuticals, Cambridge, MA, USA.
White MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Adams D; Neurology Department, APHP, CHU Bicêtre, Université Paris-Saclay, U1195, INSERM, Neurology Department, AP-HP, CHU Bicêtre, Le Kremlin Bicêtre, France.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2023 Mar; Vol. 30 (1), pp. 49-58. Date of Electronic Publication: 2022 Sep 18.
Typ publikacji:
Journal Article
MeSH Terms:
Polyneuropathies*/drug therapy
Amyloid Neuropathies, Familial*/drug therapy
Humans ; Quality of Life ; Treatment Outcome ; Biomarkers ; Prealbumin
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Liver-directed drugs for transthyretin-mediated amyloidosis.
Autorzy:
Brannagan TH 3rd; Peripheral Neuropathy Center, Columbia University, Vagelos College of Physicians and Surgeons, New York, New York, USA.
Berk JL; Amyloidosis Center, Boston University School of Medicine, Boston, Massachusetts, USA.
Gillmore JD; National Amyloidosis Centre, University College London, Royal Free Hospital, London, UK.
Maurer MS; Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York, New York, USA.
Waddington-Cruz M; National Amyloidosis Referral Center-CEPARM, University Hospital, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Fontana M; National Amyloidosis Centre, University College London, Royal Free Hospital, London, UK.
Masri A; Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health & Science University, Portland, Oregon, USA.
Obici L; Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy.
Brambatti M; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Baker BF; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Hannan LA; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Buchele G; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Viney NJ; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Coelho T; Department of Neurosciences, Centro Hospitalar Universitário do Porto, Porto, Portugal.
Nativi-Nicolau J; Division of Heart Failure and Transplant, Mayo Clinic, Jacksonville, Florida, USA.
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Źródło:
Journal of the peripheral nervous system : JPNS [J Peripher Nerv Syst] 2022 Dec; Vol. 27 (4), pp. 228-237. Date of Electronic Publication: 2022 Nov 16.
Typ publikacji:
Journal Article; Review; Research Support, Non-U.S. Gov't
MeSH Terms:
Amyloid Neuropathies, Familial*/drug therapy
Amyloid Neuropathies, Familial*/genetics
Prealbumin*/genetics
Prealbumin*/metabolism
Humans ; Liposomes/therapeutic use ; Liver/metabolism ; RNA, Messenger/genetics ; RNA, Small Interfering/therapeutic use
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis.
Autorzy:
Ando Y; Department of Amyloidosis Research, Nagasaki International University, Sasebo, Japan.
Adams D; Department of Neurology, French National Reference Centre for Familial Amyloidotic Polyneuropathy, CHU Bicêtre, Université Paris-Saclay, Le Kremlin-Bicêtre, France.
Benson MD; Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN, USA.; RLR Veterans Affairs Medical Center, Indianapolis, IN, USA.
Berk JL; Amyloidosis Center, Boston Medical Center, Boston University, Boston, MA, USA.
Planté-Bordeneuve V; Department of Neurology and Amyloid Network, Hospital Henri Mondor, APHP, East-Paris University, Créteil, France.
Coelho T; Andrade's Center, Centro Hospitalar Univerisitário do Porto - Hospital de Santo António, Porto, Portugal.
Conceição I; Department of Neurosciences and Mental Health, CHULN - Hospital de Santa Maria and Faculdade de Medicina, Instituto de Fisiologia, Universidade de Lisboa, Lisbon, Portugal.
Ericzon BG; Division of Transplantation Surgery, Karolinska University Hospital Huddinge, Stockholm, Sweden.
Obici L; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy.
Rapezzi C; Cardiologic Center, University of Ferrara, and Maria Cecilia Hospital, GVM Care & Research, Cotignola, Italy.
Sekijima Y; Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan.
Ueda M; Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan.
Palladini G; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy.
Merlini G; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2022 Sep; Vol. 29 (3), pp. 143-155. Date of Electronic Publication: 2022 Jun 02.
Typ publikacji:
Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial*/drug therapy
Amyloid Neuropathies, Familial*/genetics
Cardiomyopathies*/drug therapy
Cardiomyopathies*/genetics
Liver Transplantation*
Humans ; Prealbumin/genetics
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis.
Autorzy:
Aimo A; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.; Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Rapezzi C; Cardiology Division, University Hospital of Ferrara, Ferrara, Italy.; Maria Cecilia Hospital, Cotignola, Italy.
Perfetto F; Regional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Cappelli F; Regional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Palladini G; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.; Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.; Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Merlini G; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.; Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Di Bella G; Cardiology Division, University of Messina, Messina, Italy.
Serenelli M; Cardiology Division, University Hospital of Ferrara, Ferrara, Italy.; Maria Cecilia Hospital, Cotignola, Italy.
Zampieri M; Regional Amyloid Centre, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Milani P; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.; Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Licordari R; Cardiology Division, University of Messina, Messina, Italy.
Teresi L; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Ribarich N; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Castiglione V; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Quattrone F; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.; Fondazione Toscana Gabriele Monasterio, Pisa, Italy.; Management and Healthcare Laboratory, Institute of Management, Scuola Superiore Sant'Anna, Pisa, Italy.
De Rosis S; Management and Healthcare Laboratory, Institute of Management, Scuola Superiore Sant'Anna, Pisa, Italy.
Vergaro G; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.; Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Panichella G; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.
Emdin M; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.; Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
Passino C; Institute of Life Sciences, Scuola Superiore Sant'Anna, Pisa, Italy.; Fondazione Toscana Gabriele Monasterio, Pisa, Italy.
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Źródło:
European journal of clinical investigation [Eur J Clin Invest] 2021 Nov; Vol. 51 (11), pp. e13598. Date of Electronic Publication: 2021 May 22.
Typ publikacji:
Journal Article; Review
MeSH Terms:
Quality of Life*
Amyloid Neuropathies/*physiopathology
Amyloid Neuropathies, Familial/*physiopathology
Cardiomyopathies/*physiopathology
Humans ; Patient Reported Outcome Measures
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy.
Autorzy:
Garcia-Pavia P; Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, CIBERCV, Madrid, Spain.; Universidad Francisco de Vitoria (UFV), Pozuelo de Alarcon, Spain.; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart-ERN GUARD-Heart, Amsterdam, The Netherlands.
Bengel F; Department of Nuclear Medicine, Hannover Medical School, Hannover, Germany.
Brito D; Heart and Vessels Department, Centro Hospitalar Universitário de Lisboa Norte, CCUL, Faculdade de Medicina da Universidade de Lisboa, Lisbon, Portugal.
Damy T; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart-ERN GUARD-Heart, Amsterdam, The Netherlands.; Referral Center for Cardiac Amyloidosis, GRC Amyloid Research Institute, Department of Cardiology, Centre Hospitalier Universitaire Henri Mondor, DHU-ATVB Créteil, France and Inserm U955, Université Paris-Est Créteil (UPEC), Créteil, France.
Duca F; Department of Internal Medicine II, Division of Cardiology, Medical University of Vienna, Vienna, Austria.
Dorbala S; Division of Nuclear Medicine and Molecular Imaging, Department of Radiology, Brigham and Women's Hospital, Boston, MA, USA.
Nativi-Nicolau J; Department of Medicine, University of Utah Health Care, Salt Lake City, UT, USA.
Obici L; Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy.
Rapezzi C; Cardiological Centre, University of Ferrara, Ferrara, Italy.; Maria Cecilia Hospital, GVM Care & Research, Cotignola, Italy.
Sekijima Y; Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan.
Elliott PM; University College London Institute for Cardiovascular Science & St Bartholomew's Hospital, London, UK.
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Źródło:
European journal of heart failure [Eur J Heart Fail] 2021 Jun; Vol. 23 (6), pp. 895-905. Date of Electronic Publication: 2021 May 24.
Typ publikacji:
Journal Article; Research Support, Non-U.S. Gov't; Review
MeSH Terms:
Amyloid Neuropathies, Familial*/diagnosis
Cardiomyopathies*/diagnosis
Heart Failure*
Consensus ; Humans ; Prealbumin/genetics
Czasopismo naukowe
Tytuł:
ATTRv amyloidosis Italian Registry: clinical and epidemiological data.
Autorzy:
Russo M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Obici L; IRCCS Fondazione Policlinico San Matteo, University of Pavia, Amyloidosis Research and Treatment Center, Pavia, Italy.
Bartolomei I; Amyloidosis Centre, University of Bologna, Bologna, Italy.
Cappelli F; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Luigetti M; Fondazione Policlinico Universitario A. Gemelli IRCCS. UOC Neurologia, Rome, Italy.
Fenu S; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Cavallaro T; Department of Neurosciences, Biomedicine and Movement Sciences, University of Verona, Verona, Italy.
Chiappini MG; Fatebenefratelli Foundation-'San Giovanni Calibita' Fatebenefratelli Hospital, Clinical Pathophysiology Center, Rome, Italy.
Gemelli C; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health (DINOGMI), University of Genoa, Genoa, Italy.
Pradotto LG; San Giuseppe Hospital, IRCCS-Istituto Auxologico Italiano, Division of Neurology and Neurorehabilitation, Piancavallo, Italy.; Department of Neuroscience, University of Turin, Turin, Italy.
Manganelli F; Department of Neurosciences Reproductive and Odontostomatological Sciences, University Federico II, Naples, Italy.
Leonardi L; Unit of Neuromuscular Diseases, Department of Neurology, Mental Health and Sensory Organs (NESMOS), Sapienza University, Sant'Andrea Hospital, Rome, Italy.
My F; Division of Neurology, Ospedale V. Fazzi, Lecce, Italy.
Sampaolo S; Second Division of Neurology, Department of Advanced Medical and Surgical Sciences, University of Campania Luigi Vanvitelli, Naples, Italy.
Briani C; Department of Neuroscience, Neurology Unit, University of Padova School of Medicine and Surgery, Padova, Italy.
Gentile L; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Stancanelli C; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Di Buduo E; IRCCS Fondazione Policlinico San Matteo, University of Pavia, Amyloidosis Research and Treatment Center, Pavia, Italy.
Pacciolla P; IRCCS Fondazione Policlinico San Matteo, University of Pavia, Amyloidosis Research and Treatment Center, Pavia, Italy.
Salvi F; Amyloidosis Centre, University of Bologna, Bologna, Italy.
Casagrande S; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Bisogni G; Centro Clinico NEMO adulti - Roma, Rome, Italy.
Calabrese D; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Vanoli F; Unit of Neuromuscular Diseases, Department of Neurology, Mental Health and Sensory Organs (NESMOS), Sapienza University, Sant'Andrea Hospital, Rome, Italy.
Di Iorio G; Second Division of Neurology, Department of Advanced Medical and Surgical Sciences, University of Campania Luigi Vanvitelli, Naples, Italy.
Antonini G; Unit of Neuromuscular Diseases, Department of Neurology, Mental Health and Sensory Organs (NESMOS), Sapienza University, Sant'Andrea Hospital, Rome, Italy.
Santoro L; Department of Neurosciences Reproductive and Odontostomatological Sciences, University Federico II, Naples, Italy.
Mauro A; San Giuseppe Hospital, IRCCS-Istituto Auxologico Italiano, Division of Neurology and Neurorehabilitation, Piancavallo, Italy.; Department of Neuroscience, University of Turin, Turin, Italy.
Grandis M; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health (DINOGMI), University of Genoa, Genoa, Italy.
Di Girolamo M; Fatebenefratelli Foundation-'San Giovanni Calibita' Fatebenefratelli Hospital, Clinical Pathophysiology Center, Rome, Italy.
Fabrizi GM; Department of Neurosciences, Biomedicine and Movement Sciences, University of Verona, Verona, Italy.
Pareyson D; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Sabatelli M; Fondazione Policlinico Universitario A. Gemelli IRCCS. UOC Neurologia, Rome, Italy.
Perfetto F; Department of Internal and Experimental Medicine, University of Florence, Florence, Italy.
Rapezzi C; Department of Cardiology, University of Ferrara, Ferrara, Italy.; Maria Cecilia Hospital GVM, Care and Research, Cotignola, Italy.
Merlini G; IRCCS Fondazione Policlinico San Matteo, University of Pavia, Amyloidosis Research and Treatment Center, Pavia, Italy.
Mazzeo A; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Vita G; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2020 Dec; Vol. 27 (4), pp. 259-265. Date of Electronic Publication: 2020 Jul 22.
Typ publikacji:
Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial/*epidemiology
Adult ; Aged ; Aged, 80 and over ; Amyloid Neuropathies, Familial/diagnosis ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/pathology ; Cardiomyopathies/epidemiology ; Cardiomyopathies/pathology ; Female ; Genotype ; Humans ; Italy/epidemiology ; Male ; Middle Aged ; Mutation ; Phenotype ; Polyneuropathies/epidemiology ; Polyneuropathies/pathology ; Prealbumin/genetics ; Prevalence ; Registries
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Description of a large cohort of Caucasian patients with V122I ATTRv amyloidosis: Neurological and cardiological features.
Autorzy:
Gentile L; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Di Bella G; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Minutoli F; Department of Biomedical and Dental Sciences and Morphofunctional Imaging, University of Messina, Messina, Italy.
Cucinotta F; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Mussinelli R; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Arimatea I; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Russo M; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Toscano A; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Vita G; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Mazzeo A; Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
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Źródło:
Journal of the peripheral nervous system : JPNS [J Peripher Nerv Syst] 2020 Sep; Vol. 25 (3), pp. 273-278. Date of Electronic Publication: 2020 May 29.
Typ publikacji:
Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial*/complications
Amyloid Neuropathies, Familial*/diagnosis
Amyloid Neuropathies, Familial*/ethnology
Amyloid Neuropathies, Familial*/genetics
Heart Diseases*/diagnosis
Heart Diseases*/ethnology
Heart Diseases*/etiology
Heart Diseases*/genetics
Polyneuropathies*/diagnosis
Polyneuropathies*/ethnology
Polyneuropathies*/etiology
Polyneuropathies*/genetics
White People*/ethnology
White People*/genetics
Prealbumin/*genetics
Adult ; Aged ; Follow-Up Studies ; Humans ; Male ; Middle Aged ; Sicily/ethnology
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
Autorzy:
Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Berk JL; Amyloidosis Center, Boston Medical Center, Boston, MA, USA.
González-Duarte A; Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Coelho T; Hospital de Santo António, Centro Hospitalar do Porto, Porto, Portugal.
Gillmore J; Division of Medicine, National Amyloidosis Centre, University College London, London, UK.
Schmidt HH; Medical Clinic for Gastroenterology and Hepatology, University of Münster, Münster, Germany.
Schilling M; Department of Neurology, Institute of Translational Neurology, University Hospital Münster, Münster, Germany.
Yamashita T; Department of Neurology, Kumamoto University, Kumamoto, Japan.
Labeyrie C; Assistance Publique-Hôpitaux de Paris (APHP), French National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire Bicêtre, Universite Paris-Sud, INSERM Unite, Paris, France.
Brannagan TH 3rd; Department of Neurology, Columbia University Medical Center, New York, NY, USA.
Ajroud-Driss S; Department of Neurology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
Gorevic P; Department of Medicine, Mount Sinai Medical Center, New York, NY, USA.
Kristen AV; Department of Cardiology, University of Heidelberg, Heidelberg, Germany.
Franklin J; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Chen J; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Wang JJ; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Adams D; Assistance Publique-Hôpitaux de Paris (APHP), French National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire Bicêtre, Universite Paris-Sud, INSERM Unite, Paris, France.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2020 Sep; Vol. 27 (3), pp. 153-162. Date of Electronic Publication: 2020 Mar 04.
Typ publikacji:
Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial
MeSH Terms:
Amyloid Neuropathies, Familial/*drug therapy
Polyneuropathies/*drug therapy
Prealbumin/*genetics
RNA, Small Interfering/*administration & dosage
Adolescent ; Adult ; Aged ; Aged, 80 and over ; Amyloid Neuropathies, Familial/complications ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/pathology ; Female ; Humans ; Male ; Middle Aged ; Mutation/drug effects ; Placebo Effect ; Polyneuropathies/complications ; Polyneuropathies/genetics ; Polyneuropathies/pathology ; Quality of Life ; Young Adult
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Plasma neurofilament light chain: an early biomarker for hereditary ATTR amyloid polyneuropathy.
Autorzy:
Maia LF; Molecular Neurobiology Group, Instituto de Biologia Molecular e Celular (IBMC) - Instituto de Investigação e Inovação em Saúde (i3S) da Universidade do Porto, Porto, Portugal.; Neurology Department, Centro Hospitalar Universitário do Porto (CHUP), Porto, Portugal.; Instituto de Ciências Biomédicas Abel Salazar (ICBAS), Universidade do Porto, Porto, Portugal.
Maceski A; Neurologic Clinic and Policlinic, Departments of Medicine, Biomedicine and Clinical Research, University Hospital Basel, University of Basel, Basel, Switzerland.
Conceição I; Neurology Department, Centro Hospitalar Universitário Lisboa Norte (CHULN) and Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.
Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Magalhães R; Instituto de Ciências Biomédicas Abel Salazar (ICBAS), Universidade do Porto, Porto, Portugal.
Cortese A; Department of Brain and Behavioral Sciences, University of Pavia, Pavia, Italy.
Leppert D; Neurologic Clinic and Policlinic, Departments of Medicine, Biomedicine and Clinical Research, University Hospital Basel, University of Basel, Basel, Switzerland.
Merlini G; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Kuhle J; Neurologic Clinic and Policlinic, Departments of Medicine, Biomedicine and Clinical Research, University Hospital Basel, University of Basel, Basel, Switzerland.
Saraiva MJ; Molecular Neurobiology Group, Instituto de Biologia Molecular e Celular (IBMC) - Instituto de Investigação e Inovação em Saúde (i3S) da Universidade do Porto, Porto, Portugal.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2020 Jun; Vol. 27 (2), pp. 97-102. Date of Electronic Publication: 2020 Jan 06.
Typ publikacji:
Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial/*diagnosis
Biomarkers/*metabolism
Intermediate Filaments/*metabolism
Adult ; Aged ; Aged, 80 and over ; Amyloid/metabolism ; Amyloid Neuropathies, Familial/blood ; Biomarkers/blood ; Cross-Sectional Studies ; Female ; Humans ; Male ; Middle Aged ; Mutation ; Retrospective Studies
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression.
Autorzy:
Conceição I; a CHLN-Hospital Santa Maria, IMM, Unversidade de Lisboa , Lisbon , Portugal.
Coelho T; b Unidade Corino de Andrade, Hospital de Santo António, Centro Hospitalar Universitário do Porto , Porto , Portugal.
Rapezzi C; c Department of Experimental, Diagnostic and Specialty Medicine, University of Bologna , Bologna , Italy.
Parman Y; d Neurology Department, Neuromuscular Unit, Istanbul Faculty of Medicine, Istanbul University , Istanbul , Turkey.
Obici L; e IRCCS Fondazione Policlinico San Matteo, Amyloidosis Research and Treatment Center , Pavia , Italy.
Galán L; f Department of Neurology, Hospital Clinico San Carlos IdiSSC , Madrid , Spain.
Rousseau A; g Department of Ophthalmology, Bicêtre Hospital Université Paris-Sud, Le Kremlin-Bicêtre, France APHP, DHU Sight Restore, French Reference centre for H-ATTR (NNERF), French Reference Network for Rare Ophthalmic Diseases (OPHTARA) , Le Kremlin-Bicêtre , France.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2019 Sep; Vol. 26 (3), pp. 103-111. Date of Electronic Publication: 2019 Jul 24.
Typ publikacji:
Journal Article; Review
MeSH Terms:
Disease Management*
Amyloid Neuropathies, Familial/*diagnosis
Cardiomyopathies/*diagnosis
Glaucoma/*diagnosis
Hereditary Sensory and Autonomic Neuropathies/*diagnosis
Adult ; Age of Onset ; Aged ; Amyloid Neuropathies, Familial/drug therapy ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/physiopathology ; Cardiomyopathies/drug therapy ; Cardiomyopathies/genetics ; Cardiomyopathies/physiopathology ; Consensus ; Disease Progression ; Female ; Glaucoma/drug therapy ; Glaucoma/genetics ; Glaucoma/physiopathology ; Heart Function Tests ; Hereditary Sensory and Autonomic Neuropathies/drug therapy ; Hereditary Sensory and Autonomic Neuropathies/genetics ; Hereditary Sensory and Autonomic Neuropathies/physiopathology ; Humans ; Kidney Function Tests ; Male ; Middle Aged ; Mutation ; Neuroprotective Agents/therapeutic use ; Prealbumin/deficiency ; Prealbumin/genetics
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Transthyretin deposition in the eye in the era of effective therapy for hereditary ATTRV30M amyloidosis.
Autorzy:
Buxbaum JN; a The Scripps Research Institute , La Jolla , CA, USA.
Brannagan T 3rd; b Department of Neurology , NewYork-Presbyterian Hospital , New York , NY , USA.
Buades-Reinés J; c Department of Internal Medicine , Son Llatzer Hospital , Palma de Mallorca , Spain.
Cisneros E; d Department of Cardiology , Son Llatzer Hospital , Palma de Mallorca , Spain.
Conceicao I; e Department of Neurosciences , Centro Hospitalar Lisboa Norte-Hospital Santa Maria , Lisbon , Portugal.; f IIM, Faculdade de Medicina de Lisboa , Lisbon , Portugal.
Kyriakides T; g Cyprus Instirute of Neurology and Genetics , Nicosia , Cyprus.
Merlini G; h Department of Molecular Medicine , University of Pavia, Policlinico San Matteo , Pavia , Italy.
Obici L; i Fondazione IRCCS Policlinico San Matteo, Amyloid Research and Treatment Center , Biotechnology Research Laboratories , Pavia , Italy.
Plante-Bordeneuve V; j Department of Neurology , Hopital Nenri Mondor , Creteil , France.
Rousseau A; k Department of Ophthalmology, French Reference Center for familial transthyretin amyloidosis (NNERF), French Reference Network for rare Ophthalmologic diseases (OPHTARA) , Bicêtre Hospital, Assistance Publique - Hôpitaux de Paris, DHU Vision & Handicaps, Paris-Sud , Le Kremlin-Bicêtre , France.
Sekijima Y; l Department of Medicine (Neurology & Rheumatology), Shinshu University School of Medicine , Matsumoto , Japan.
Imai A; m Department of Opthalmology , Shinshu University School of Medicine , Matsumoto , Japan.
Waddington Cruz M; n Department of Neurology, Federal University of Rio de Janeiro, Rua Professor Rodolpho Paulo Rocco , Rio de Janeiro , Brazil.
Yamada M; o Department of Neurology & Neurobiology of Aging , Kanazawa University Graduate School of Medical Science , Kanazawa , Japan.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2019 Mar; Vol. 26 (1), pp. 10-14. Date of Electronic Publication: 2019 Jan 24.
Typ publikacji:
Journal Article; Multicenter Study
MeSH Terms:
Mutation, Missense*
Protein Aggregation, Pathological*
Amyloid Neuropathies, Familial/*drug therapy
Benzoxazoles/*therapeutic use
Eye Diseases/*drug therapy
Prealbumin/*genetics
Amyloid Neuropathies, Familial/complications ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/metabolism ; Benzoxazoles/pharmacology ; Eye Diseases/etiology ; Eye Diseases/genetics ; Eye Diseases/metabolism ; Humans ; Prealbumin/drug effects ; Prealbumin/metabolism
Czasopismo naukowe
Tytuł:
Progressive axonal polyneuropathy in a mitochondrial disorder: an uncommon association with familial amyloid neuropathy.
Autorzy:
Luigetti M; a Fondazione Policlinico Universitario A. Gemelli IRCCS , UOC Neurologia, Roma , Italia.; b Università Cattolica del Sacro Cuore , Roma , Italia.
Primiano G; c Fondazione Policlinico Universitario A. Gemelli IRCCS , UOC Neurofisiopatologia, Roma , Italia.
Bisogni G; d Centro Clinico NEMO adulti , Roma , Italia.
Cuccagna C; e Fondazione Policlinico Universitario A. Gemelli IRCCS , UOC Neurologia, Roma , Italia.
Carrozzo R; f Unit of Neuromuscular and Neurodegenerative Disorders , Bambino Gesù Children's Research Hospital , Rome , Italy.
Obici L; g Amyloidosis Research and Treatment Center , Foundation IRCCS Policlinico San Matteo, Pavia , Italy.
Bernardo D; h Centro Clinico NEMO adulti , Roma , Italia.
Sancricca C; i Fondazione Policlinico Universitario A. Gemelli IRCCS , UOC Neurologia, Roma , Italia.
Servidei S; j Università Cattolica del Sacro Cuore , Roma , Italia.
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2018 Dec; Vol. 25 (4), pp. 261-262. Date of Electronic Publication: 2018 Sep 07.
Typ publikacji:
Case Reports; Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial/*pathology
Mitochondrial Diseases/*complications
Aged ; Amyloid Neuropathies, Familial/etiology ; Humans ; Male ; Mitochondrial Diseases/physiopathology ; Prognosis
Czasopismo naukowe
Tytuł:
Differential expression of Cathepsin E in transthyretin amyloidosis: from neuropathology to the immune system.
Autorzy:
Gonçalves NP; i3S - Instituto de Investigação e Inovação em Saúde da Universidade do Porto, Rua Alfredo Allen, 208, 4200-135, Porto, Portugal.; Molecular Neurobiology Group, IBMC - Institute for Molecular and Cell Biology, University of Porto, 4150-180, Porto, Portugal.; Present address: Department of Biomedicine/DANDRITE, Aarhus University, Ole Worms Alle 3, 1171, 8000, Aarhus C, Denmark.
Moreira J; i3S - Instituto de Investigação e Inovação em Saúde da Universidade do Porto, Rua Alfredo Allen, 208, 4200-135, Porto, Portugal.; Molecular Neurobiology Group, IBMC - Institute for Molecular and Cell Biology, University of Porto, 4150-180, Porto, Portugal.
Martins D; i3S - Instituto de Investigação e Inovação em Saúde da Universidade do Porto, Rua Alfredo Allen, 208, 4200-135, Porto, Portugal.; Molecular Neurobiology Group, IBMC - Institute for Molecular and Cell Biology, University of Porto, 4150-180, Porto, Portugal.
Vieira P; Unité du Développement des Lymphocytes, Département d'Immunologie, Institut Pasteur, Paris, 75724 CEDEX 15, France.
Obici L; Amyloidosis Research and Treatment Center, Department of Molecular Medicine, Fondazione Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, University of Pavia, Pavia, Italy.
Merlini G; Amyloidosis Research and Treatment Center, Department of Molecular Medicine, Fondazione Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, University of Pavia, Pavia, Italy.
Saraiva M; i3S - Instituto de Investigação e Inovação em Saúde da Universidade do Porto, Rua Alfredo Allen, 208, 4200-135, Porto, Portugal.; Immune Regulation Group, IBMC - Institute for Molecular and Cell Biology, University of Porto, 4150-180, Porto, Portugal.
Saraiva MJ; i3S - Instituto de Investigação e Inovação em Saúde da Universidade do Porto, Rua Alfredo Allen, 208, 4200-135, Porto, Portugal. .; Molecular Neurobiology Group, IBMC - Institute for Molecular and Cell Biology, University of Porto, 4150-180, Porto, Portugal. .
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Źródło:
Journal of neuroinflammation [J Neuroinflammation] 2017 Jun 06; Vol. 14 (1), pp. 115. Date of Electronic Publication: 2017 Jun 06.
Typ publikacji:
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:
Amyloid Neuropathies, Familial/*genetics
Amyloid Neuropathies, Familial/*immunology
Cathepsin E/*genetics
Cathepsin E/*immunology
Immunity, Cellular/*immunology
Adult ; Amyloid Neuropathies, Familial/pathology ; Animals ; Cathepsin E/biosynthesis ; Female ; Gene Expression ; Humans ; Male ; Mice ; Mice, Transgenic ; Middle Aged
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
"Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy.
Autorzy:
Conceição I; CHLN - Hospital de Santa Maria, and Clinical and Translational Physiology Unit, Faculty of Medicine-IMM, Physiology Institute, Lisbon, Portugal.
González-Duarte A; Department of Neurology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, México City, México.
Obici L; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Schmidt HH; Klinik für Transplantationsmedizin, Universitätsklinikum Münster, Münster, Germany.
Simoneau D; Medical Division, Pfizer International Operations, Paris, France.
Ong ML; Global Medical Affairs, Global Innovative Pharma, Pfizer Inc, New York, NY, USA.
Amass L; Global Medical Affairs, Global Innovative Pharma, Pfizer Inc, New York, NY, USA.
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Źródło:
Journal of the peripheral nervous system : JPNS [J Peripher Nerv Syst] 2016 Mar; Vol. 21 (1), pp. 5-9.
Typ publikacji:
Journal Article; Research Support, Non-U.S. Gov't; Review
MeSH Terms:
Early Diagnosis*
Amyloid Neuropathies, Familial/*diagnosis
Humans
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
TTR-related amyloid neuropathy: clinical, electrophysiological and pathological findings in 15 unrelated patients.
Autorzy:
Luigetti M; Institute of Neurology, Catholic University of Sacred Heart, Largo F. Vito 1, 00168 Rome, RM, Italy. />Conte A
Del Grande A
Bisogni G
Madia F
Lo Monaco M
Laurenti L
Obici L
Merlini G
Sabatelli M
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Źródło:
Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology [Neurol Sci] 2013 Jul; Vol. 34 (7), pp. 1057-63. Date of Electronic Publication: 2012 May 17.
Typ publikacji:
Journal Article
MeSH Terms:
Amyloid Neuropathies, Familial/*diagnosis
Amyloid Neuropathies, Familial/*genetics
Mutation/*genetics
Prealbumin/*genetics
Aged ; Amyloid/metabolism ; Amyloid Neuropathies/diagnosis ; Amyloid Neuropathies/genetics ; Amyloid Neuropathies/physiopathology ; Amyloid Neuropathies, Familial/epidemiology ; Amyloid Neuropathies, Familial/physiopathology ; Female ; Genetic Testing ; Humans ; Italy/epidemiology ; Male ; Middle Aged
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
High 99mTc-DPD myocardial uptake in a patient with apolipoprotein AI-related amyloidotic cardiomyopathy.
Autorzy:
Quarta CC; Institute of Cardiology, University of Bologna and S. Orsola-Malpighi Hospital, Bologna, Italy.
Obici L
Guidalotti PL
Pieroni M
Longhi S
Perlini S
Verga L
Merlini G
Rapezzi C
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2013 Mar; Vol. 20 (1), pp. 48-51. Date of Electronic Publication: 2012 Dec 12.
Typ publikacji:
Case Reports; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:
Amyloid Neuropathies, Familial/*metabolism
Apolipoprotein A-I/*genetics
Cardiomyopathies/*metabolism
Diphosphonates/*metabolism
Organotechnetium Compounds/*metabolism
Radiopharmaceuticals/*metabolism
Amyloid Neuropathies, Familial/complications ; Amyloid Neuropathies, Familial/diagnosis ; Amyloid Neuropathies, Familial/diagnostic imaging ; Cardiomyopathies/complications ; Cardiomyopathies/diagnosis ; Cardiomyopathies/diagnostic imaging ; Humans ; Male ; Middle Aged ; Mutation ; Myocardium/metabolism ; Myocardium/pathology ; Radionuclide Imaging
SCR Disease Name:
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł:
Guideline of transthyretin-related hereditary amyloidosis for clinicians.
Autorzy:
Ando Y; Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, 1-1-1 Honjo, Chuo-ku, Kumamoto, 860-8556, Japan. />Coelho T
Berk JL
Cruz MW
Ericzon BG
Ikeda S
Lewis WD
Obici L
Planté-Bordeneuve V
Rapezzi C
Said G
Salvi F
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Źródło:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2013 Feb 20; Vol. 8, pp. 31. Date of Electronic Publication: 2013 Feb 20.
Typ publikacji:
Journal Article; Review
MeSH Terms:
Practice Guidelines as Topic*
Practice Patterns, Physicians'*
Amyloid Neuropathies, Familial/*therapy
Prealbumin/*metabolism
Age of Onset ; Amyloid Neuropathies, Familial/epidemiology ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/metabolism ; Humans ; Prealbumin/genetics ; Prevalence
Czasopismo naukowe
Tytuł:
Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study.
Autorzy:
Obici L; Amyloid Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy. />Cortese A
Lozza A
Lucchetti J
Gobbi M
Palladini G
Perlini S
Saraiva MJ
Merlini G
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Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2012 Jun; Vol. 19 Suppl 1, pp. 34-6. Date of Electronic Publication: 2012 May 02.
Typ publikacji:
Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:
Amyloid Neuropathies, Familial/*drug therapy
Amyloid Neuropathies, Familial/*genetics
Doxycycline/*therapeutic use
Prealbumin/*genetics
Taurochenodeoxycholic Acid/*therapeutic use
Adult ; Aged ; Drug Administration Schedule ; Female ; Humans ; Male ; Middle Aged ; Treatment Outcome
Czasopismo naukowe
Tytuł:
The Diflunisal Trial: study accrual and drug tolerance.
Autorzy:
Berk JL; Department of Medicine & Data Coordinating Center, Boston University Medical Center, Boston, MA 02118, USA. />Suhr OB
Sekijima Y
Yamashita T
Heneghan M
Zeldenrust SR
Ando Y
Ikeda S
Gorevic P
Merlini G
Kelly JW
Skinner M
Bisbee AB
Dyck PJ
Obici L
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Corporate Authors:
Familial Amyloidosis Consortium
Źródło:
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis [Amyloid] 2012 Jun; Vol. 19 Suppl 1, pp. 37-8. Date of Electronic Publication: 2012 May 02.
Typ publikacji:
Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
MeSH Terms:
Amyloid Neuropathies, Familial/*drug therapy
Anti-Inflammatory Agents, Non-Steroidal/*therapeutic use
Diflunisal/*therapeutic use
Adult ; Aged ; Anti-Inflammatory Agents, Non-Steroidal/adverse effects ; Diflunisal/adverse effects ; Female ; Humans ; Male ; Middle Aged ; Young Adult
Czasopismo naukowe

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