Gene therapy offers hope for kids with rare brain disorder
NCT ID NCT02852213
First seen Nov 01, 2025 · Last updated Apr 24, 2026 · Updated 29 times
Summary
This study tests a gene therapy for children with AADC deficiency, a rare genetic disorder that affects movement and brain function. The treatment delivers a working gene directly to the brain using a virus. The goal is to see if it is safe and can improve symptoms like muscle control and reduce episodes of eye spasms. The study is in its early phase and is currently recruiting children aged 2 and older.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
Contact
Contact Email: •••••@•••••
Contact
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The Ohio State University Medical Center
RECRUITINGColumbus, Ohio, 43221, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact
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University of California San Francisco, Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
Contact Email: •••••@•••••
Contact
Contact
Conditions
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