Dentatorubral-pallidoluysian atrophy
Clinical trials for Dentatorubral-pallidoluysian atrophy explained in plain language.
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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 …
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
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…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
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…
Matched conditions: DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC