Allan-Herndon-Dudley syndrome
MONDO:0010354A syndrome with neuromuscular involvement characterized by infantile hypotonia, muscular hypoplasia, spastic paraparesis with dystonic/athetoic movements, and severe cognitive deficiency.
Also known as: AHDS, ALLAN-Herndon syndrome, Allan-Herndon-Dudley syndrome, MCT8 deficiency, MCT8-Specific Thyroid Hormone Cell Transporter Deficiency, MCT8-specific thyroid hormone cell Membrane transporter deficiency, X-linked intellectual disability-hypotonia syndrome, monocarboxylate transporter 8 deficiency
6 clinical trials for this condition and its sub-types, 6 tagged with Allan-Herndon-Dudley syndrome itself.
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New hope for rare brain disorder: early drug access program opens
Disease control Expanded accessThis program offers early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Patients must have a confirmed genetic diagnosis and be considered likely to be…
Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Experimental drug offers hope for babies with rare genetic brain condition
Disease control Expanded accessThis trial tests a drug called DITPA in male infants with MCT8 deficiency, a rare genetic disorder that causes severe intellectual disability and movement problems. The drug aims to improve thyroid hormone function in the brain. Only infants whose families have a history of the c…
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC