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Trials to join now! 1

One-Time IV treatment aims to halt fatal childhood brain disease

Disease control Recruiting now

This study is testing a one-time intravenous (IV) gene therapy for children with GM1 gangliosidosis, a rare and fatal genetic disorder that destroys nerve cells. The therapy aims to deliver a working copy of a missing gene so the body can produce a vital enzyme, potentially slowi…

Matched conditions: GM1

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

Last updated Mar 31, 2026 12:12 UTC

GM1

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One-Time IV treatment aims to halt fatal childhood brain disease