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.

Dominique P. Germain, Roberto Giugliani, Derralynn A. Hughes, Atul Mehta, Kathy Nicholls, Laura Barisoni, Charles J. Jennette, Alexander Bragat, Jeff Castelli, Sheela Sitaraman, David J. Lockhart, Pol F. Boudes

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77 Scopus citations

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