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, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 wee…
Matched conditions: VANISHING WHITE MATTER DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Calico Life Sciences LLC • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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Major study tracks rare brain diseases to unlock their secrets
Knowledge-focused Recruiting nowThis study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC