VANISHING WHITE MATTER DISEASE
Clinical trials for VANISHING WHITE MATTER DISEASE explained in plain language.
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New drug trial offers hope for rare brain disease
Disease control Recruiting nowThis study tests an experimental drug called fosigotifator in people with Vanishing White Matter disease, a rare genetic brain disorder. About 50 adults, children, and infants will receive the drug to see if it is safe and how the body processes it. Researchers will also look for…
Matched conditions: VANISHING WHITE MATTER DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Calico Life Sciences LLC • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 11:05 UTC
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Scientists watch rare brain diseases unfold in 1,500 patients
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 26, 2026 10:35 UTC