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Olipudase alfa versus placebo in adults with acid sphingomyelinase deficiency
NL-OMON54584Phase 2Recruiting
- Sponsor
- Sanofi
- Start
- Oct 2016
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in acid sphingomyelinase deficiency (ASMD) and Niemann-Pick disease type B (NPD-B) comparing Olipudase alfa with Placebo (2 arms). Primary endpoints: Spleen volume and Pulmonary function. Started Oct 2016; status: recruiting.
02
Drugs
olipudase alfa
03
Conditions
acid sphingomyelinase deficiency (ASMD)Niemann-Pick disease type B (NPD-B)
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