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Prospective observational study of adults with late-onset Pompe disease on alglucosidase alfa
NL-OMON46633Phase 4Not yet recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Sep 2018
- 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 Amicus Therapeutics. Primary endpoints: 6-minute walk test, Forced vital capacity and 26 more. Planned start Sep 2018; status: not yet recruiting.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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