Rescue of Infants With MCT8 Deficiency

Summary

Monocarboxylate Transporter 8 (MCT8) deficiency (that is also known as Allan-Herndon-Dudley syndrome) is a rare X-linked inherited disorder of brain development that causes severe intellectual disability and problems with movement. This condition, which occurs almost exclusively in males, disrupts development from before birth.

Timeline

Start

—

Primary completion

—

Completion

—

Drugs

Indications