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

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

Completed
Phase of Trial: Phase I/II

Latest Information Update: 19 May 2014

At a glance

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

    • 19 May 2014 New trial record
    • 05 Apr 2007
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