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Avalglucosidase alfa ascending-dose study in infantile-onset Pompe disease with suboptimal response to alglucosidase alfa
2016-003475-21Phase 2Unknown
- Sponsor
- Sanofi
- Start
- Jan 2017
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoint: Number of participants with adverse events. Status: unknown.
02
Drugs
avalglucosidase alfa
03
Conditions
Pompe disease
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