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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Groundbreaking prenatal treatment trial for devastating Boy's disease
Disease control AVAILABLEThis study is testing a treatment for a rare genetic disorder called MCT8 deficiency, which causes severe intellectual and movement disabilities in boys. The treatment is given to the mother during pregnancy, with the goal of helping the baby's brain develop more normally. The st…
Matched conditions: MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated Mar 12, 2026 13:50 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC