ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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New hope for rare genetic disorder: early access to tiratricol
Disease control AVAILABLEThis program provides early access to tiratricol for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Participants must have a confirmed genetic diagnosis and may be new to tiratricol or switching from another…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 13, 2026 16:02 UTC