Leukodystrophy
MONDO:0019046Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the brain, spinal cord and often the peripheral nerves. Each type of leukodystrophy is caused by a specific gene abnormality that leads to abnormal development or destruction of the white matter (myelin sheath) of the brain. The myelin sheath is the protective covering of the nerve and nerves can't function normally without it. Each type of leukodystrophy affects a different part of the myelin sheath, leading to a range of neurological problems.
Also known as: hypomyelinating leukodystrophy, hypomyelinating leukoencephalopathy, leukodystrophy, hypomyelinating
68 clinical trials for this condition and its sub-types.
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Broader categories
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Can a repurposed drug slow rare childhood brain diseases?
Disease control Not yet recruitingThis phase 2 trial is testing an oral drug called PLX-200 (gemfibrozil) in children aged 2 to 15 with certain lysosomal storage disorders (LSDs), including CLN2, CLN3, Sandhoff disease, and Krabbe disease. The study aims to see if the drug is safe, tolerable, and may slow the pro…
Phase: PHASE2 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
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Could a simple gel boost gum healing in diabetes?
Disease control Not yet recruitingThis study tests whether adding hyaluronic acid gel to standard deep cleaning (scaling and root planing) improves gum healing in people with both diabetes and advanced gum disease. Twenty-three participants will receive the standard cleaning on all teeth, and then two gum pockets…
Phase: NA • Sponsor: Marmara University • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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Robot suit aims to get kids with disabilities walking
Symptom relief Not yet recruitingThis study tests a robotic exoskeleton called CLINICAL EXPLORER in 60 children aged 2 to 17 with neurodevelopmental disorders like cerebral palsy. The device supports walking during 8 therapy sessions. Researchers will check if it is safe, easy to use, and helps improve movement …
Phase: NA • Sponsor: MarsiBionics • Aim: Symptom relief
Last updated Jun 26, 2026 14:47 UTC
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Could pomegranate supplements help heal the gut in Alcohol-Related liver disease?
Knowledge-focused Not yet recruitingThis study investigates how pomegranate dietary supplements influence gut bacteria and inflammation in people with alcohol use disorder and alcohol-related liver disease. Researchers will measure levels of beneficial gut metabolites called urolithins and inflammatory markers in b…
Sponsor: University of Louisville • Aim: Knowledge-focused
Last updated Jul 26, 2026 00:00 UTC