One-of-a-Kind genetic treatment trial launched for Ultra-Rare disorder
NCT ID NCT07197268
Summary
This is a study for a single child with Bainbridge-Ropers syndrome, a rare genetic disorder that causes severe developmental delays. Researchers are testing a custom-made genetic drug designed specifically for the child's unique gene variant. The main goals are to see if the treatment is safe and if it can help improve the child's motor skills, communication, and quality of life over two years.
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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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