A Phase 2 study of migalastat hydrochloride in females with Fabry disease: Selection of population, safety and pharmacodynamic effects
Under a Creative Commons license
open access
Keywords
Fabry disease
Lysosomal storage disorders
Pharmacological chaperone
α-Galactosidase A
Globotriaosylceramide
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Retired from Salford Royal NHS Foundation Trust October 2011.
Copyright © 2013 Elsevier Inc. Published by Elsevier Inc.