One-of-a-Kind drug trial targets rare genetic brain disease
NCT ID NCT07221760
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disorder. The treatment aims to reduce seizures and improve quality of life. Only one participant is enrolled, and the study tracks changes in seizure activity and caregiver burden over two years.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
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Contacts and locations
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Locations
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Dell Children's
Austin, Texas, 78723, United States
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