Gene therapy in the womb: a new hope for babies with fatal brain disease
NCT ID NCT07479953
First seen Mar 20, 2026 · Last updated Jun 12, 2026 · Updated 13 times
Summary
This study tests a one-time gene therapy given directly to the fetus through the mother's bloodstream before birth. It is for babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The goal is to see if the treatment is safe and can help control the disease by providing a working copy of the missing enzyme. Only 5 participants will be enrolled.
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This is a summary of
the original study
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.