Clinical Trial Update: A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 [Phase 3]

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Summary

Phase 3 multinational trial of venglustat for late-onset GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) was terminated; results have been posted.

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Diseases / indications

• Sandhoff Disease
• Tay-sachs Disease

Drugs mentioned

• Venglustat (venglustat)

Snippet

Sponsor: Genzyme, a Sanofi Company | Conditions: Tay-Sachs Disease, Sandhoff Disease | Phase 3 | Status: TERMINATED | Results posted: 2026-01-28