Custom drug targets rare brain disorder in first-of-its-kind trial
NCT ID NCT07221760
First seen Nov 01, 2025 · Last updated May 11, 2026 · Updated 28 times
Summary
This study tests a custom-made genetic drug (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disease. The drug aims to reduce seizures and improve quality of life. Only one participant is enrolled, and the study is in early phases (1/2) to check safety and effects.
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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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Dell Children's
Austin, Texas, 78723, United States
Conditions
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