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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Pioneering prenatal treatment aims to rescue babies from devastating genetic disorder
Disease control AVAILABLEThis study tests whether starting treatment before birth and continuing after delivery can improve brain development in male infants with MCT8 deficiency, a rare genetic disorder that causes severe intellectual disability and movement problems. The research involves giving a thyr…
Matched conditions: MCT8 (SLC16A2)-SPECIFIC THYROID HORMONE CELL TRANSPORTER DEFICIENCY
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated Apr 03, 2026 14:42 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