ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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Experimental drug offered to patients with rare, severe genetic disorder
Disease control AVAILABLEThis program provides access to an experimental drug called tiratricol for patients with Allan-Herndon-Dudley syndrome, a rare and severe genetic disorder that affects brain development and movement. Eligible patients who cannot join clinical trials can receive this treatment bef…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Mar 23, 2026 15:14 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC