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Venglustat versus Cerezyme in pediatric Gaucher disease type 3
2026-525374-19-00Phase 3Authorised
- Sponsor
- Sanofi
- Start
- Sep 2026
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 14
01
Overview
Phase 3, randomised, open-label trial in Gaucher disease type 3 comparing Venglustat with Cerezyme (2 arms). Primary endpoint: Percent change from baseline in MRI spleen volume. Planned start Sep 2026; status: authorised.
02
Drugs
venglustatimiglucerase
03
Conditions
Gaucher disease type 3
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Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
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