GM1 GANGLIOSIDOSES

Clinical trials for GM1 GANGLIOSIDOSES explained in plain language.

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Trials to join now! 1 Not yet recruiting 1

First-ever prenatal gene therapy trial aims to stop deadly brain disease before birth

Disease control Not yet recruiting

This early-stage study tests a one-time gene therapy given directly to the fetus before birth for GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The treatment uses a harmless virus to deliver a working copy of the missing enzyme. The main goal is t…

Matched conditions: GM1 GANGLIOSIDOSES

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

Last updated May 11, 2026 20:53 UTC

GM1 GANGLIOSIDOSES

Get alerted when new GM1 GANGLIOSIDOSES trials appear

First-ever prenatal gene therapy trial aims to stop deadly brain disease before birth