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Prospective study of adults with late-onset Pompe disease receiving alglucosidase alfa enzyme replacement therapy
2017-004370-34Phase 4Terminated
- Sponsor
- Amicus Therapeutics
- Start
- Feb 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 endpoint: Change in key clinical outcome measures. Started Feb 2018; status: terminated.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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