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Avalglucosidase alfa in treatment-naive infants with infantile-onset Pompe disease
NL-OMON60419Phase 3Completed
- Sponsor
- Sanofi
- Start
- Aug 2022
- 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: Alive and free of invasive ventilation at Week 52. Started Aug 2022; status: completed.
02
Drugs
avalglucosidase alfa
03
Conditions
Pompe disease
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