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Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

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 investigation...

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Detalles Bibliográficos
Autores principales:	Germain, Dominique P, Giugliani, Roberto, Hughes, Derralynn A, Mehta, Atul, Nicholls, Kathy, Barisoni, Laura, Jennette, Charles J, Bragat, Alexander, Castelli, Jeff, Sitaraman, Sheela, Lockhart, David J, Boudes, Pol F
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2012
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3527132/ https://www.ncbi.nlm.nih.gov/pubmed/23176611 http://dx.doi.org/10.1186/1750-1172-7-91

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