DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Clinical trials for DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY explained in plain language.
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Custom drug targets rare genetic brain disease in one patient
Disease control OngoingThis 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. The study is early-…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 26, 2026 12:05 UTC
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One-of-a-kind drug trial aims to slow rare brain disorder
Disease control OngoingThis 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 cha…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 20, 2026 11:56 UTC