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Avalglucosidase alfa in treatment-naive pediatric participants with infantile-onset Pompe disease
2020-004686-39Phase 3Active
- Sponsor
- Sanofi
- Start
- Jan 2021
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoint: Proportion of participants alive and free of invasive ventilation at Week 52. Started Jan 2021; status: active.
02
Drugs
avalglucosidase alfa
03
Conditions
Pompe disease
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