Clinical Trial Update: Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support [Phase 3]

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Summary

A Phase 3 trial evaluating rhGAA (alglucosidase alfa) in patients with advanced late-onset Pompe disease on respiratory support has completed, assessing safety …

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Diseases / indications

• Glycogen Storage Disease Type Ii
• Pompe Disease

Drugs mentioned

• Myozyme (alglucosidase alfa)

Snippet

Sponsor: Genzyme, a Sanofi Company | Conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II) | Phase 3 | Status: COMPLETED