MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Clinical trials for MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY explained in plain language.
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Breakthrough trial offers hope for babies with rare brain disorder
Disease control AVAILABLEThis study aims to help male infants with MCT8 deficiency, a rare genetic condition that causes severe intellectual and movement problems. It offers early treatment with thyroid hormone to improve brain development. Participants are babies whose families have chosen not to end th…
Matched conditions: MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated May 26, 2026 08:34 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 08:32 UTC