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Long-term open-label extension of cipaglucosidase alfa + miglustat in adults with late-onset Pompe disease
2023-505170-15-00Phase 3Authorised
- Sponsor
- Amicus Therapeutics
- Start
- May 2020
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 19
01
Overview
Phase 3, open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoint: Long-term safety. Planned start May 2020; status: authorised.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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