TrialGenetics in medicine : official journal of the American College of Medical Genetics2019

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 et al.PubMed ↗Full text ↗Publisher ↗

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