VANISHING WHITE MATTER DISEASE
Clinical trials for VANISHING WHITE MATTER DISEASE explained in plain language.
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First drug trial launched for devastating brain disease in children and adults
Disease control Recruiting nowThis study is testing an experimental drug called fosigotifator for vanishing white matter disease, a rare genetic brain disorder that affects both children and adults. Researchers will give the drug to about 50 participants over 201 weeks to check if it's safe and how the body p…
Matched conditions: VANISHING WHITE MATTER DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Calico Life Sciences LLC • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: VANISHING WHITE MATTER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC