Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Dominique P. Germain(Assistance Publique – Hôpitaux de Paris), Christopher Viereck(Université Paris-Saclay), Laura Barisoni(Duke University), Elfrida R. Benjamin(Université Paris-Saclay), Kathy Nicholls(Melbourne Health), J. Charles Jennette(University of North Carolina at Chapel Hill), Nina Skuban(Université Paris-Saclay), Daniel G. Bichet(Hôpital du Sacré-Cœur de Montréal), Roberto Giugliani(Universidade Federal do Rio Grande do Sul), Jeffrey P. Castelli(Amicus Therapeutics (United States)), Derralynn Hughes(Royal Free London NHS Foundation Trust), Jay Barth(Université Paris-Saclay), Robert B. Colvin(Harvard University)
Genetics in Medicine
February 5, 2019
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