First gene therapy trial for rare brain disorder launches
NCT ID NCT07293546
First seen Jan 11, 2026 · Last updated May 25, 2026 · Updated 26 times
Summary
This early-stage trial tests a single dose of FRF-001 gene therapy in 12 children and adults with FOXG1 syndrome, a rare genetic condition affecting brain development. The therapy is injected directly into the brain's fluid spaces to deliver a working copy of the FOXG1 gene. Researchers will monitor safety and check if participants gain new motor skills, like sitting or crawling.
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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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Conditions
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