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Long-term follow-up of olipudase alfa in ASMD patients from DFI12712 or LTS13632 (France)
2024-515304-39-00Phase 2Completed
- Sponsor
- Sanofi
- Start
- Aug 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of olipudase alfa in acid sphingomyelinase deficiency (ASMD) and Niemann-Pick disease type A, sponsored by Sanofi. Primary endpoint: Safety and tolerability of olipudase alfa. Started Aug 2024; status: completed.
02
Drugs
olipudase alfa
03
Conditions
acid sphingomyelinase deficiency (ASMD)Niemann-Pick disease type A
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