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 Paul GermainKathy NichollsRoberto GiuglianiDaniel G BichetDerralynn A HughesLaura M BarisoniRobert B ColvinJ Charles JennetteNina SkubanJeffrey P CastelliElfrida BenjaminJay A BarthChristopher Viereck

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2019)

Migalastat provided clinical benefit to patients with Fabry disease and amenable variants, regardless of disease severity.

Keyphrases

double blind
clinical trial
placebo controlled
phase iii
phase ii
copy number
replacement therapy
open label
study protocol
hypertrophic cardiomyopathy
gene expression
randomized controlled trial
heart failure
dna methylation
heat shock protein
left ventricular
genome wide