Groundbreaking prenatal treatment trial for devastating Boy's disease
Disease control
AVAILABLE
This 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…
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated Mar 12, 2026 13:50 UTC