Allan-herndon-dudley syndrome
Clinical trials for Allan-herndon-dudley syndrome explained in plain language.
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New hope for rare brain disorder: early drug access program opens
Disease control Expanded accessThis program offers early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Patients must have a confirmed genetic diagnosis and be considered likely to be…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 27, 2026 12:29 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: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC