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High-dose or increased-frequency Myozyme in Pompe disease with suboptimal response
2015-000582-31Phase 4
- Sponsor
- Sanofi
- Start
- Apr 2015
- 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: Efficacy response during treatment versus baseline for participants with respiratory decline on standard treatment.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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