One-of-a-kind drug trial aims to slow rare brain disorder
NCT ID NCT06706388
First seen Apr 25, 2026 · Last updated May 20, 2026 · Updated 5 times
Summary
This study tests a personalized medicine for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disease that affects movement and thinking. The drug is an antisense oligonucleotide designed to target the faulty ATN1 gene. Researchers will track changes in walking, coordination, seizures, and quality of life over two years to see if the treatment helps control the disease.
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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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