Brain gene therapy offers hope for kids with rare movement disorder
NCT ID NCT02852213
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 31 times
Summary
This study tests a one-time gene therapy for children with AADC deficiency, a rare genetic disorder that causes severe movement problems and developmental delays. The therapy delivers a working gene directly to the brain using a harmless virus. The goal is to improve motor function and reduce symptoms like eye-rolling episodes. The study 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: •••••@•••••
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Contact
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University of California San Francisco, Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
Contact Email: •••••@•••••
Contact
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Conditions
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