GM1 GANGLIOSIDOSIS

Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.

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Trials to join now! 2 Not yet finished but already full! 2 Completed 2 Terminated 1

Hope fades: trial of Tay-Sachs drug venglustat terminated early

Disease control Terminated

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

Matched conditions: GM1 GANGLIOSIDOSIS

Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control

Last updated Jun 27, 2026 09:00 UTC

GM1 GANGLIOSIDOSIS

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Hope fades: trial of Tay-Sachs drug venglustat terminated early