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12,372 trials

Phase 3 N=100 Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Glycogen Storage Disease Type II;Pompe's Disease
Primary: PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 — 2.89; 0.46 percent predicted FVC — p=0.0074
Genzyme, a Sanofi Company Results Apr 2021 24.5% serious AE View details