ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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Rare disease patients get early access to promising treatment
Disease control AVAILABLEThis program provides early access to tiratricol for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects movement and development. Participants must have a confirmed genetic diagnosis and may be new to tiratricol or switching from a related stu…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 15, 2026 18:55 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