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Avalglucosidase alfa exploratory study in previously treated late-onset Pompe disease
NL-OMON56788Phase 4Recruiting
- Sponsor
- Erasmus MC
- Start
- Oct 2022
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Erasmus MC. Primary endpoints: Manual muscle testing, Hand-held dynamometry and 11 more. Started Oct 2022; status: recruiting.
02
Drugs
avalglucosidase alfa
03
Conditions
Pompe disease
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