Clinical Trial Update: A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease [Phase 2, Phase 3]

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Summary

Phase 2/3 trials evaluated the safety and efficacy of recombinant human acid alpha-glucosidase (alglucosidase alfa) in infants with Pompe disease; the studies a…

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Diseases / indications

• Pompe Disease

Drugs mentioned

• Myozyme (alglucosidase alfa)

Snippet

Sponsor: Genzyme, a Sanofi Company | Conditions: Glycogen Storage Disease Type II | Phase 2, Phase 3 | Status: COMPLETED