First-ever prenatal gene therapy trial aims to stop deadly brain disease before birth
NCT ID NCT07479953
First seen Mar 20, 2026 · Last updated May 09, 2026 · Updated 7 times
Summary
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 to check safety for both mother and baby, with only 5 participants planned.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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