AICARDI GOUTIERES SYNDROME
Clinical trials for AICARDI GOUTIERES SYNDROME explained in plain language.
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Scientists launch study to unravel RNA's role in rare brain diseases
Knowledge-focused Recruiting nowThis study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…
Matched conditions: AICARDI GOUTIERES SYNDROME
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated Jun 22, 2026 12:05 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: AICARDI GOUTIERES SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 16, 2026 12:53 UTC