GM1 gangliosidosis, type I

Clinical trials for GM1 gangliosidosis, type I explained in plain language.

Never miss a new study

Get alerted when new GM1 gangliosidosis, type I trials appear

Sign up with your email to follow new studies for GM1 gangliosidosis, type I, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1

Gene therapy before birth: a new hope for babies with GM1 disease

Disease control Not yet recruiting

This early-stage trial tests giving a gene therapy to unborn babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The therapy uses a harmless virus to deliver a working copy of the missing enzyme gene. The main goal is to see if the…

Matched conditions: GM1 GANGLIOSIDOSIS, TYPE I

Phase 1 • Sponsor: Tippi Mackenzie • Aim: Disease control

Last updated Sep 02, 2026 00:00 UTC

GM1 gangliosidosis, type I

Get alerted when new GM1 gangliosidosis, type I trials appear

Gene therapy before birth: a new hope for babies with GM1 disease