First gene therapy for rare brain disorder begins testing in kids
NCT ID NCT07270549
First seen Jan 07, 2026 · Last updated May 22, 2026 · Updated 22 times
Summary
This study tests a new gene therapy for children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic disorder causing severe developmental delays. Twelve participants will receive a single dose of the therapy directly into the brain fluid, along with lifelong immune-suppressing drugs to prevent rejection. The goal is to see if it is safe and can improve motor skills, thinking, behavior, sleep, and quality of life over three years.
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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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Genom att skicka in godkänner du våra Användarvillkor
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Genom att skicka in godkänner du våra Användarvillkor
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Phone: •••-•••-•••• Email: •••••@•••••
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Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University Medical Centre Ljubljana
RECRUITINGLjubljana, 1000, Slovenia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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