Tay-Sachs disease

MONDO:0010100

GM2 gangliosidosis, variant B or Tay-Sachs disease is marked by accumulation of G2 gangliosides due to hexosaminidase A deficiency.

Also known as: GM2 gangliosidosis, B, B1 variant, GM2-gangliosidosis, several forms, Hex A pseudodeficiency, Tay Sachs Disease, Tay-Sachs disease, disease, Tay-Sachs, hexosaminidase A deficiency, B variant GM2 gangliosidosis

19 clinical trials for this condition and its sub-types, 13 tagged with Tay-Sachs disease itself.

Follow this condition to get notified about new trials

Where it sits in the disease tree

Browse by category →

Part of

↑ Eye disorder (3079) ↑ Hereditary peripheral neuropathy (479) ↑ Cerebral lipidosis with dementia (38) ↑ GM2 gangliosidosis (19)

Sub-types of Tay-Sachs disease

Tay-Sachs disease, B variant, adult form 3 trials
Tay-Sachs disease, b variant, juvenile form 1 trial
Tay-Sachs disease, B1 variant 0 trials
Tay-Sachs disease, b variant, infantile form 0 trials

Including sub-types (19) Tagged with Tay-Sachs disease (13)

Trials to join now! 8 Not yet recruiting 1 Not yet finished but already full! 2 Completed 4 Terminated 4

New gene therapy hopes to fix fatal brain diseases

Disease control Not yet recruiting

This study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, fix the genetic defect in the lab, and then see if these cells can help brain cells work better. The goal is to show the …

Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control

Last updated Jun 27, 2026 09:11 UTC

Tay-Sachs disease

Where it sits in the disease tree

New gene therapy hopes to fix fatal brain diseases