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Miglustat added to enzyme replacement therapy in Pompe disease
2010-024647-32Phase 2Active
- Sponsor
- University of Naples Federico II
- Start
- May 2011
- Modality
- Small Molecules, Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of miglustat and alglucosidase alfa in Pompe disease, sponsored by University of Naples Federico II. Primary endpoints: Peak acid alpha-glucosidase activity and Serum creatine phosphokinase reduction. Started May 2011; status: active.
02
Drugs
miglustatalglucosidase alfa
03
Conditions
Pompe disease
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