GM1 gangliosidosis

MONDO:0018149

A rare lysosomal storage disorder characterized biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable neurovisceral, ophthalmological and dysmorphic features.

Also known as: Beta-galactosidase-1 deficiency, GLB1 deficiency, GM>1< gangliosidosis, Landing disease, Landing syndrome, gangliosidosis GM1, Beta galactosidase 1 deficiency, Beta-galactosidosis

12 clinical trials for this condition and its sub-types, 12 tagged with GM1 gangliosidosis itself.

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Where it sits in the disease tree

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Part of

↑ Bone disorder (6305) ↑ Eye disorder (3079) ↑ Gangliosidosis (24)

Sub-types of GM1 gangliosidosis

Including sub-types (12) Tagged with GM1 gangliosidosis (12)

Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 3 Completed 2 Terminated 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

GM1 gangliosidosis

Where it sits in the disease tree

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