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 offers early access to tiratricol for people with Allan-Herndon-Dudley syndrome, a rare genetic disorder that affects movement and development. Participants must have a confirmed genetic diagnosis and may be new to tiratricol or already taking it from a previous stud…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Apr 30, 2026 15:49 UTC
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Massive biorepository aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical information and biological samples from people with leukodystrophy, a group of rare brain disorders. Researchers will use this data to find new genetic causes, develop better tests, and understand how these diseases progress. T…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 01, 2026 16:01 UTC