Dentatorubral-pallidoluysian atrophy
MONDO:0007435Dentatorubral pallidoluysian atrophy (DRPLA) is a rare subtype of type I autosomal dominant cerebellar ataxia (ADCA type I). It is characterized by involuntary movements, ataxia, epilepsy, mental disorders, cognitive decline and prominent anticipation.
Also known as: DRPLA, Dentatorubropallidoluysian atrophy, Naito-Oyanagi disease, dentatorubral-pallidoluysian atrophy, haw River syndrome, NOD, Naito Oyanagi disease, ataxia, chorea, seizures, and dementia
23 clinical trials for this condition and its sub-types, 4 tagged with Dentatorubral-pallidoluysian atrophy itself.
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Custom-Made genetic drug targets rare brain disease in first human test
Disease control OngoingThis study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The treatment aims to reduce seizures and improve quality of life by targeting the …
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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Custom drug targets rare genetic brain disease in First-Ever human test
Disease control OngoingThis study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated AT…
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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One-of-a-Kind drug trial targets rare genetic brain disease
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. Only one participan…
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC