Experimental drug offers hope for babies with rare genetic brain condition
Disease control
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This trial tests a drug called DITPA in male infants with MCT8 deficiency, a rare genetic disorder that causes severe intellectual disability and movement problems. The drug aims to improve thyroid hormone function in the brain. Only infants whose families have a history of the c…
Sponsor: Roy E. Weiss, M.D. • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC