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Open-label phase 3 study of cipaglucosidase alfa + miglustat in pediatric infantile-onset Pompe disease
MOH_2023-09-14_012671Phase 3Not yet recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Aug 2023
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label trial of cipaglucosidase alfa and miglustat in Pompe disease (2 cohorts), sponsored by Amicus Therapeutics. Planned start Aug 2023; status: not yet recruiting.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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