One-person trial aims to slow rare brain disease with custom genetic drug
NCT ID NCT07222371
First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 28 times
Summary
This study tests an experimental genetic medicine (antisense oligonucleotide) in a single person with TUBB4A-related leukodystrophy, a rare brain disorder that affects movement. The goal is to see if the treatment can improve or stabilize motor function and reduce dystonia (involuntary muscle contractions). The participant will be closely monitored over time using standard movement and behavior scales.
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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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Rady Children's Hospital San Diego
San Diego, California, 92123, United States
Conditions
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