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
Germain, Giugliani Roberto, Hughes, Mehta Atul, Nicholls Kathy, Barisoni Laura, Jennette, Bragat Alexander, Castelli Jeff, Sitaraman Sheela, Lockhart, Boudes