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Phase 3 open-label extension of ATB200 + AT2221 in adults with late-onset Pompe disease
NL-OMON54552Phase 3Recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Nov 2020
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoints: Long-term safety profile and Immunogenicity to ATB200. Started Nov 2020; status: recruiting.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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