GANGLIOSIDOSIS

Clinical trials for GANGLIOSIDOSIS explained in plain language.

Never miss a new study

Get alerted when new GANGLIOSIDOSIS trials appear

Sign up with your email to follow new studies for GANGLIOSIDOSIS, 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

Experimental gene therapy offers hope for children with fatal GM1 disease

Disease control Recruiting now

This study tests a one-time gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1…

Matched conditions: GANGLIOSIDOSIS

Phase: PHASE1, PHASE2 • Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control

Last updated May 26, 2026 08:19 UTC

GANGLIOSIDOSIS

Get alerted when new GANGLIOSIDOSIS trials appear

Experimental gene therapy offers hope for children with fatal GM1 disease