ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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Experimental drug tiratricol now available for rare genetic disorder before official approval
Disease control AVAILABLEThis program provides early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects movement and development. Eligible patients must have a confirmed genetic diagnosis and a doctor who believes…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 23, 2026 11:59 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 16, 2026 12:53 UTC