One-of-a-kind drug trial aims to slow rare brain disease
NCT ID NCT06706388
First seen Apr 25, 2026 · Last updated May 17, 2026 · Updated 4 times
Summary
This study tests a custom-made drug (called an antisense oligonucleotide) designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The drug targets the faulty ATN1 gene to potentially slow or improve movement problems, seizures, and quality of life. Only one participant is enrolled, and researchers will track changes 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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Contacts and locations
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Locations
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Columbia University
New York, New York, 10027, United States
Conditions
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