An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease

Status: Completed

Phase of Trial: Phase II/III

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; Registrational; Therapeutic Use
Sponsors Genzyme Corporation

Most Recent Events

19 May 2014 New trial record
24 Jul 2006

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