Familial thyroid dyshormonogenesis
MONDO:0010132A type of primary congenital hypothyroidism, a permanent thyroid hormone deficiency that is present from birth, which results from inborn errors of thyroid hormone synthesis.
Also known as: dyshormonogenesis, nongoitrous hyperthyrotropinemia, thyroid dyshormonogenesis
4 clinical trials for this condition and its sub-types.
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Broader categories
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Stem cell hope for rare brain disease: new expanded access trial opens
Disease control AVAILABLEThis study offers expanded access to an experimental stem cell treatment for up to 7 adults with Multiple System Atrophy (MSA), a rare and serious brain disorder. Participants will receive 12 intravenous infusions and 6 spinal injections of donor stem cells over 44 weeks. The goa…
Sponsor: Hope Biosciences Research Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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New protein therapy hopes to repair brain damage in rare disease
Disease control Recruiting nowThis early-phase trial tests a drug called Aleeto, a nerve repair protein made from stem cells, in 20 people with Multiple System Atrophy (MSA), a rare brain disease. The study checks if Aleeto is safe and might help with symptoms. Participants receive the drug via injection into…
Phase: EARLY_PHASE1 • Sponsor: Beijing Tiantan Hospital • Aim: Disease control
Last updated Jun 26, 2026 12:56 UTC
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Blood test breakthrough could spot Parkinson's before symptoms worsen
Diagnosis Recruiting nowThis study is testing a new blood test to help doctors diagnose Parkinson's disease and similar brain disorders more accurately and earlier. Researchers will use a technique called RT-QuIC to detect misfolded proteins in blood samples from 458 participants, including healthy peop…
Sponsor: Xuanwu Hospital, Beijing • Aim: Diagnosis
Last updated Jun 27, 2026 12:01 UTC