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Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
Journal of Medical Genetics · 2016 · Vol. 54(4) · pp. 288–296
Derralynn Hughes✉(Royal Free London NHS Foundation Trust)Kathy Nicholls(The Royal Melbourne Hospital)Suma P. Shankar(Emory University)Gere Sunder‐Plassmann(Medical University of Vienna)David M. Koeller(Doernbecher Children's Hospital)Khan Nedd(Infusion Associates)Jerry Vockley(Children's Hospital of Pittsburgh)Takashi Hamazaki(Osaka City University Hospital)Robin Lachmann(University College London)Toya Ohashi(The Jikei University Hospital)Iacopo Olivotto(Azienda Ospedaliero-Universitaria Careggi)Norio Sakai(Osaka City University Hospital)Patrick Deegan(Addenbrooke's Hospital)David Dimmock(Children's Hospital of Wisconsin)François EyskensDominique P. Germain(Université Paris-Saclay)Özlem Göker-Alpan(O&O Alpan (United States))É. Hachulla(Hôpital Claude Huriez)Ana Jovanović(Salford Royal NHS Foundation Trust)Charles Marques Lourenço(Clinics Hospital of Ribeirão Preto)Ichiei Narita(Niigata University)Mark Thomas(Royal Perth Hospital)William R. Wilcox(Emory University)Daniel G. Bichet(Hôpital du Sacré-Cœur de Montréal)Raphael SchiffmannElizabeth LudingtonChristopher Viereck(Amicus Therapeutics (United States))John Kirk(Amicus Therapeutics (United States))Julie Yu(Amicus Therapeutics (United States))Franklin K. Johnson(Amicus Therapeutics (United States))Pol Boudes(CymaBay Therapeutics (United States))Elfrida R. Benjamin(Amicus Therapeutics (United States))David J. LockhartCarrolee Barlow(Parkinson's Institute and Clinical Center)Nina Skuban(Amicus Therapeutics (United States))Jeffrey P. Castelli(Amicus Therapeutics (United States))Jay Barth(Amicus Therapeutics (United States))Ulla Feldt‐Rasmussen(Copenhagen University Hospital)
Abstract
NCT00925301; Pre-results.
Lysosomal Storage Disorders ResearchTrypanosoma species research and implicationsGlycogen Storage Diseases and MyoclonusFabry diseaseEnzyme replacement therapyMedicineInternal medicineBioinformaticsPharmacologyDiseaseBiology
MeSH terms
Administration, OralAdolescentAdultAgedalpha-GalactosidaseFabry DiseaseFemaleHumansLysosomesMaleMiddle AgedTreatment Outcome1-DeoxynojirimycinMolecular ChaperonesEnzyme Replacement Therapy
Funding
- Amicus Therapeutics
Citations
380
FWCI
19.47
field-weighted impact
References
60
Percentile
100%
vs. same field & year
Citations per year
References
High Incidence of Later-Onset Fabry Disease Revealed by Newborn Screening*
The American Journal of Human Genetics · 2006 · 997 citations
Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy
Nephrology Dialysis Transplantation · 2009 · 363 citations
A New Equation to Estimate Glomerular Filtration Rate
Annals of Internal Medicine · 2009 · 25,291 citations
The MOS 36-ltem Short-Form Health Survey (SF-36)
Medical Care · 1992 · 29,431 citations
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