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Phase 2 dose-comparison study of recombinant human acid sphingomyelinase in acid sphingomyelinase deficiency
2010-023953-12Phase 2Completed
- Sponsor
- Sanofi
- Start
- Jul 2013
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, randomised, open-label trial of olipudase alfa in Niemann-Pick disease type B (NPD-B) and acid sphingomyelinase deficiency (ASMD) (2 arms), sponsored by Sanofi. Primary endpoint: Percentage change in spleen volume. Started Jul 2013; status: completed.
02
Drugs
olipudase alfa
03
Conditions
Niemann-Pick disease type B (NPD-B)acid sphingomyelinase deficiency (ASMD)
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