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Wyszukujesz frazę ""Mulberg, Andrew"" wg kryterium: Autor


Tytuł:
Correction to: Sharing is caring: a call for a new era of rare disease research and development
Autorzy:
Denton, NathanAff1, IDs13023023026139_cor1
Mulberg, Andrew E.
Molloy, Monique
Charleston, Samantha
Fajgenbaum, David C.Aff3, Aff4
Marsh, Eric D.Aff3, Aff5, Aff6, IDs13023023026139_cor6
Howard, PaulAff7, IDs13023023026139_cor7
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Źródło:
Orphanet Journal of Rare Diseases. 18(1)
Czasopismo naukowe
Tytuł:
Sharing is caring: a call for a new era of rare disease research and development
Autorzy:
Denton, NathanAff1, Aff7, IDs1302302202529w_cor1
Mulberg, Andrew E.
Molloy, Monique
Charleston, Samantha
Fajgenbaum, David C.Aff3, Aff4
Marsh, Eric D.Aff3, Aff5, Aff6, Aff7, IDs1302302202529w_cor6
Howard, PaulAff7, IDs1302302202529w_cor7
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Źródło:
Orphanet Journal of Rare Diseases. 17(1)
Czasopismo naukowe
Tytuł:
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Autorzy:
Shields AL; Adelphi Values, 290 Congress Street, 6th Floor, Boston, MA, 02210, USA. .
Lamoureux RE; Adelphi Values, 290 Congress Street, 6th Floor, Boston, MA, 02210, USA.
Taylor F; Adelphi Values, 290 Congress Street, 6th Floor, Boston, MA, 02210, USA.
Barth JA; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Mulberg AE; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Kessler V; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Skuban N; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
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Źródło:
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation [Qual Life Res] 2021 Oct; Vol. 30 (10), pp. 2983-2994. Date of Electronic Publication: 2021 Apr 29.
Typ publikacji:
Journal Article
MeSH Terms:
Fabry Disease*
Diarrhea/etiology ; Humans ; Models, Theoretical ; Patient Reported Outcome Measures ; Quality of Life/psychology
Czasopismo naukowe
Tytuł:
Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy‐Induced Nausea and Vomiting.
Autorzy:
Momper, Jeremiah D.
Heinrichs, M. Tobias
Krudys, Kevin
Griebel, Donna
Kumar, Shaun
Kim, Insook
Mehrotra, Nitin
Mulberg, Andrew E.
Garimella, Narayana
Nelson, Robert
Reaman, Gregory
Sinha, Vikram
Yao, Lynne
Zineh, Issam
Burckart, Gilbert
Sachs, Hari
Mulugeta, Yeruk
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Źródło:
Journal of Clinical Pharmacology. Jun2020, Vol. 60 Issue 6, p775-784. 10p.
Czasopismo naukowe
Tytuł:
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
Autorzy:
Mulberg AE; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA. .
Bucci-Rechtweg C; Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ, 07936, USA.
Giuliano J; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Jacoby D; BioMarin Pharmaceutical Inc., 770 Lindaro St, San Rafael, CA, 94901, USA.
Johnson FK; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Liu Q; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Marsden D; Ultragenyx Pharmaceutical, Inc., 60 Leveroni Ct, Novato, CA, 94949, USA.
McGoohan S; Vertex Pharmaceuticals, 50 Northern Ave, Boston, MA, 02210, USA.
Nelson R; Johnson & Johnson, 920 Route 202 South, Raritan, NJ, 08869, USA.
Patel N; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Romero K; Critical Path Institute, 1730 East River Rd, Tucson, AZ, 85718, USA.
Sinha V; Merck & Co, Inc. 351 North Sumneytown Pike, North Wales, PA, 19454, USA.
Sitaraman S; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Spaltro J; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
Kessler V; Amicus Therapeutics, Inc., 1 Cedar Brook Drive, Cranbury, NJ, 08512, USA.
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Źródło:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2019 Feb 08; Vol. 14 (1), pp. 36. Date of Electronic Publication: 2019 Feb 08.
Typ publikacji:
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:
Rare Diseases*
Drug Approval ; Humans ; United States ; United States Food and Drug Administration
Czasopismo naukowe
Tytuł:
Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
Autorzy:
Schiffmann R; Baylor Scott & White Research Institute, Dallas, TX, USA. .; Institute of Metabolic Disease, 3812 Elm Street, Dallas, TX, 75226, USA. .
Bichet DG; Hôpital du Sacré-Coeur, University of Montreal, Montreal, Quebec, Canada.
Jovanovic A; Salford Royal Foundation Trust, Manchester, Greater Manchester, UK.
Hughes DA; NHS Foundation Trust, Royal Free Hospital, London, UK.
Giugliani R; Medical Genetics Service, HCPA/UFRGS, Porto Alegre, Brazil.
Feldt-Rasmussen U; Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Shankar SP; Emory University School of Medicine, Atlanta, GA, USA.; Present Address: UC Davis MIND Institute, Sacramento, CA, USA.
Barisoni L; Miller School of Medicine, University of Miami, Miami, FL, USA.
Colvin RB; Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Jennette JC; School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Holdbrook F; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Mulberg A; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Castelli JP; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Skuban N; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Barth JA; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
Nicholls K; Department of Nephrology, Royal Melbourne Hospital, Parkville, VIC, Australia.
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Źródło:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2018 Apr 27; Vol. 13 (1), pp. 68. Date of Electronic Publication: 2018 Apr 27.
Typ publikacji:
Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
MeSH Terms:
1-Deoxynojirimycin/*analogs & derivatives
Diarrhea/*drug therapy
Fabry Disease/*drug therapy
1-Deoxynojirimycin/therapeutic use ; Adolescent ; Adult ; Aged ; Biomarkers/metabolism ; Fabry Disease/metabolism ; Female ; Humans ; Kidney/metabolism ; Kidney/pathology ; Male ; Middle Aged ; Mutation/genetics ; Trihexosylceramides ; Young Adult
Czasopismo naukowe
Tytuł:
Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.
Autorzy:
Shapiro, Elsa
Bernstein, Jessica
Adams, Heather R.
Barbier, Ann J.
Buracchio, Teresa
Como, Peter
Delaney, Kathleen A.
Eichler, Florian
Goldsmith, Jonathan C.
Hogan, Melissa
Kovacs, Sarrit
Mink, Jonathan W.
Odenkirchen, Joanne
Parisi, Melissa A.
Skrinar, Alison
Waisbren, Susan E.
Mulberg, Andrew E. andrew.mulberg@fda.hhs.gov
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Źródło:
Molecular Genetics & Metabolism. Jun2016, Vol. 118 Issue 2, p65-69. 5p.
Czasopismo naukowe
Tytuł:
Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism.
Autorzy:
Camp, Kathryn M.
Lloyd-Puryear, Michele A.
Yao, Lynne
Groft, Stephen C.
Parisi, Melissa A.
Mulberg, Andrew Andrew.Mulberg@fda.hhs.gov
Gopal-Srivastava, Rashmi
Cederbaum, Stephen
Enns, Gregory M.
Ershow, Abby G.
Frazier, Dianne M.
Gohagan, John
Harding, Cary
Howell, R. Rodney
Regan, Karen
Stacpoole, Peter W.
Venditti, Charles
Vockley, Jerry
Watson, Michael
Coates, Paul M.
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Źródło:
Molecular Genetics & Metabolism. Aug2013, Vol. 109 Issue 4, p319-328. 10p.
Czasopismo naukowe

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