GM2 GANGLIOSIDOSIS

Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.

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Trials to join now! 4 Not yet recruiting 1 Not yet finished but already full! 1 Completed 3 Terminated 1

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 …

Matched conditions: GM2 GANGLIOSIDOSIS

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

Last updated Jun 27, 2026 09:11 UTC

GM2 GANGLIOSIDOSIS

Get alerted when new GM2 GANGLIOSIDOSIS trials appear

New gene therapy hopes to fix fatal brain diseases