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Exploratory muscle biopsy, biomarker, and imaging study in late-onset Pompe disease treated with alglucosidase alfa
2010-020611-36Phase 4Completed
- Sponsor
- Sanofi
- Start
- May 2011
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of alglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoint: Glycogen clearance in muscle tissue. Started May 2011; status: completed.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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