Clinical Trial Update: A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II [Phase 2]

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Summary

Phase 2 study evaluating the safety and pharmacokinetics of recombinant human acid alpha-glucosidase (rhGAA) in siblings with glycogen storage disease type II (…

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Diseases / indications

• Acid Maltase Deficiency Disease
• Glycogen Storage Disease Type Ii
• Glycogenosis 2
• Pompe Disease

Drugs mentioned

• LUMIZYME (ALGLUCOSIDASE ALFA)

Snippet

Sponsor: Genzyme, a Sanofi Company | Conditions: Glycogen Storage Disease Type II, Pompe Disease | Phase 2 | Status: COMPLETED