Avalglucosidase Alfa Long-term Extension in Pompe Disease

Phase 2, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoints: Number of Participants With Treatment-Emergent Adverse Events up to 458 weeks, Number of Participants With Clinically Significant Physical Examination Abnormalities up to 458 weeks and 17 more. Started Feb 2014; status: completed.