ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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Rare disease study: what happens when you stop the treatment?
Disease control CompletedThis study tested what happens when males with a rare genetic disorder called MCT8 deficiency stop taking their medication, tiratricol. For 30 days, some participants received a placebo (dummy pill) instead of their regular drug to see if their thyroid hormone levels would rise t…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Phase: PHASE3 • Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Gene test cuts diagnosis time for devastating childhood brain diseases from years to months
Diagnosis CompletedThis study tested whether using whole genome sequencing (a complete genetic map) as a first test could diagnose children with rare, inherited brain disorders called leukodystrophies faster and more accurately than the standard step-by-step approach. Researchers enrolled 236 child…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Diagnosis
Last updated Mar 25, 2026 14:07 UTC