One-person trial aims to slow rare brain disease
NCT ID NCT07222371
First seen Oct 31, 2025 · Last updated May 23, 2026 · Updated 30 times
Summary
This study tests an experimental drug called an antisense oligonucleotide in one person with TUBB4A-related leukodystrophy, a rare genetic disorder that damages the brain's white matter. The goal is to see if the treatment can improve or stabilize movement problems like dystonia (involuntary muscle contractions) and gross motor skills. The participant will be closely monitored using several standard rating scales over time.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Rady Children's Hospital San Diego
San Diego, California, 92123, United States
Conditions
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