Custom-made genetic drug tested in single patient with rare syndrome
NCT ID NCT07197268
First seen Oct 31, 2025 · Last updated May 21, 2026 · Updated 31 times
Summary
This study tests a custom-made drug (called an antisense oligonucleotide) designed for one person with Bainbridge-Ropers syndrome, a rare genetic disorder. The drug aims to correct the effects of a specific gene error. The main goals are to check if the treatment is safe and if it can improve motor skills over two years.
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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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Locations
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University of North Carolina Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Conditions
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