One-of-a-Kind genetic therapy trial launches for Ultra-Rare disorder
NCT ID NCT07197268
Summary
This study is testing a custom-made genetic medicine for a single person with a rare developmental disorder called Bainbridge-Ropers syndrome. The goal is to see if the treatment is safe and if it can help improve the participant's motor skills, communication, and quality of life. Researchers will closely monitor the person for two years to check for side effects and measure changes in their abilities.
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Contacts and locations
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Locations
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University of North Carolina Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Conditions
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