Trial Profile

An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2021

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At a glance

Drugs Alglucosidase alfa (Primary)
Indications Glycogen storage disease type II
Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Therapeutic Use
Sponsors Genzyme Corporation

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