DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Clinical trials for DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY explained in plain language.
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One-of-a-kind drug trial aims to slow rare brain disease
Disease control OngoingThis 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,…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 17, 2026 14:30 UTC
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Custom drug targets rare brain disorder in first-of-its-kind trial
Disease control OngoingThis 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 enrol…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 12, 2026 13:39 UTC