Journal article

A Phase 2 study of migalastat hydrochloride in females with Fabry disease: Selection of population, safety and pharmacodynamic effects

R Giugliani, S Waldek, DP Germain, K Nicholls, DG Bichet, JK Simosky, AC Bragat, JP Castelli, ER Benjamin, PF Boudes

Molecular Genetics and Metabolism | ACADEMIC PRESS INC ELSEVIER SCIENCE | Published : 2013