One-Time brain surgery offers hope for cure in devastating childhood disease
NCT ID NCT02852213
Summary
This study is testing a one-time gene therapy for children with AADC deficiency, a rare genetic disorder that severely impairs brain development and movement. Doctors use precise MRI-guided surgery to deliver a corrective gene directly into a specific part of the brain. The main goals are to see if this treatment is safe and if it can restore the brain's ability to make essential chemicals, potentially curing the disease's root cause.
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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Contacts and locations
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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: •••••@•••••
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The Ohio State University Medical Center
RECRUITINGColumbus, Ohio, 43221, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California San Francisco, Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
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Conditions
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