Study design & results
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Trial intelligence / Profile preview
Protocolized follow-up of Pompe disease patients receiving compassionate-use enzyme replacement therapy
NL-OMON21598Recruiting
- Sponsor
- Sanofi
- Start
- Jan 1999
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Open-label trial of alglucosidase alfa in Pompe disease (2 arms), sponsored by Sanofi. Primary endpoints: Survival and Muscle function improvement or stabilization. Started Jan 1999; status: recruiting.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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