What Does Emcitate Do for MCT8 Deficiency?
Emcitate is a daily oral tablet that lowers the toxic T3 flooding the heart, muscles, and liver of a boy born with MCT8 deficiency. In trials, it slowed the wasting and rapid heart rate that drive early death. It's the first FDA-approved drug for the disease. It doesn't cure the severe brain and motor disability that defines the syndrome, but for the body-wide part, it changes what families can do.