GM1 GANGLIOSIDOSES

Clinical trials for GM1 GANGLIOSIDOSES explained in plain language.

Never miss a new study

Get alerted when new GM1 GANGLIOSIDOSES trials appear

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

Our safety recommendation!

By submitting, you agree to our Terms of use

Trials to join now! 1 Not yet recruiting 1

Doctors test First-Ever prenatal gene therapy for devastating baby disease

Disease control Not yet recruiting

This is a first-in-human safety study testing a gene therapy given to babies before they are born. It is for fetuses diagnosed with GM1 gangliosidosis, a rare and severe genetic disorder that damages the brain and body. Doctors will inject a modified virus carrying a healthy gene…

Matched conditions: GM1 GANGLIOSIDOSES

Phase: PHASE1 • Sponsor: Tippi Mackenzie • Aim: Disease control

Last updated Mar 23, 2026 15:18 UTC

GM1 GANGLIOSIDOSES

Get alerted when new GM1 GANGLIOSIDOSES trials appear

Doctors test First-Ever prenatal gene therapy for devastating baby disease