Article
A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects.
Molecular genetics and metabolism - 1 May 2013
Giugliani R, Waldek S, Germain D P, Nicholls K, Bichet D G, Simosky J K, Bragat A C, Castelli J P, Benjamin E R, Boudes P F
Abstract excerpt
BACKGROUND: Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues. We report on the safety and pharmacodynamics of migalastat hydrochloride, an investigational pharmacological chaperone given orally every other day (QOD) to females with FD. METHODS: This was an open-label,...
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