First gene therapy trial targets rare childhood brain disorder
NCT ID NCT07173153
Summary
This is an early-stage study testing a new gene therapy for children with SLC6A1 neurodevelopmental disorder, a rare genetic condition that affects brain development. The therapy involves delivering a working copy of the faulty gene directly into the spinal fluid. The main goal is to check if this treatment is safe, while also looking for any changes in behavior and development over three years.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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