What Does Emcitate Do for MCT8 Deficiency?
Emcitate (tiratricol) is now the first FDA-approved drug for MCT8 deficiency. It's a daily oral tablet that lowers the toxic T3 flooding the heart, muscles, and liver of a boy born with this X-linked disease, and in trials it slowed the wasting and tachycardia that have driven early death. It doesn't cure the severe brain and motor disability that defines the syndrome, but it changes what families can do about the body-wide part of the disease. The earlier a family reaches diagnosis and starts dosing, the more of that benefit stays with the child.