Custom drug targets rare genetic disorder in First-Ever Single-Patient trial
NCT ID NCT07197268
First seen Oct 31, 2025 · Last updated May 11, 2026 · Updated 29 times
Summary
This study tests a custom-made genetic drug (antisense oligonucleotide) designed for one person with Bainbridge-Ropers syndrome caused by a specific ASXL3 gene change. The goal is to see if the drug is safe and can improve motor skills over two years. Because the treatment is personalized and early-stage, it focuses on safety and potential benefits for this single participant.
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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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