Genetic and Congenital

Mirum Pharmaceuticals submits new drug application for Chenodiol for CTX

Mirum Pharmaceuticals announced the submission of a New Drug Application to the U.S. Food and Drug Administration for chenodiol for the treatment of cerebrotendinous xanthomatosis (CTX), according to a press release. CTX is a rare genetic disorder affecting cholesterol metabolism, leading to severe symptoms and neurologic deterioration. The submission follows...