ALLAN-HERNDON-DUDLEY SYNDROME
Clinical trials for ALLAN-HERNDON-DUDLEY SYNDROME explained in plain language.
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MCT8 deficiency drug faces withdrawal test in small trial
Disease control CompletedThis phase 3 study tested what happens when males with MCT8 deficiency stop taking tiratricol. Twenty participants either continued tiratricol or switched to placebo for 30 days. Researchers measured thyroid hormone levels and how many needed to restart treatment. The goal was to…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Phase: PHASE3 • Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 23, 2026 11:55 UTC
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Gene sequencing breakthrough offers faster diagnosis for rare brain diseases
Diagnosis CompletedThis study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain disorders affecting white matter, more effectively than standard methods. Researchers enrolled 236 children under 18 with abnormal br…
Matched conditions: ALLAN-HERNDON-DUDLEY SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Diagnosis
Last updated Jun 15, 2026 19:07 UTC