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Tay-Sachs disease, b variant, juvenile form

MONDO:0017725

Also known as: GM2 gangliosidosis, B variant, juvenile form, hexosaminidase A deficiency, juvenile form

7 clinical trials for this condition and its sub-types, 1 tagged with Tay-Sachs disease, b variant, juvenile form itself.

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Where it sits in the disease tree

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Part of

↑ Tay-Sachs disease (13)

Including sub-types (7) Tagged with Tay-Sachs disease, b variant, juvenile form (1)

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

Disease control Stopped early

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…

Phase 3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control

Last updated Jun 27, 2026 09:00 UTC