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An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

Approved WMOPending

This study has been transitioned to CTIS with ID 2024-513859-33-00 check the CTIS register for the current data. The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and…

An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease

Approved WMORecruitment stopped

Long-term safety and pharmacokinetics (PK) of neoGAA

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease

An Open-label, Multinational, Multicenter, Intravenous
Infusion Study of the Efficacy, Safety, Pharmacokinetics,
and Pharmacodynamics of avalglucosidase alfa in
Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)