ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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Study reveals risks of stopping thyroid drug in rare genetic disorder
Disease control CompletedThis study looked at what happens when males with MCT8 deficiency (a rare genetic condition affecting thyroid hormone transport) stop taking tiratricol. Twenty participants who were stable on the drug were randomly assigned to either continue tiratricol or receive a placebo for 3…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Phase: PHASE3 • Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Apr 30, 2026 15:48 UTC
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Gene sequencing breakthrough may unlock mysterious brain diseases in kids
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete scan of a person's DNA) can help doctors diagnose leukodystrophies—rare brain disorders that damage white matter—more accurately than standard methods. About 236 children with abnormal brain MRI scans but no known g…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 17, 2026 06:52 UTC