Hope for babies with rare brain disorder: new trial offers early rescue
Disease control
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This study aims to help male infants with a rare genetic condition called MCT8 deficiency, which causes severe intellectual disability and movement problems. The trial enrolls babies with a confirmed gene mutation whose families choose not to end the pregnancy. The goal is to sta…
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated May 16, 2026 21:42 UTC