U.S. FDA Approves First Treatment for Cerebrotendinous Xanthomatosis
The U.S. Food and Drug Administration (FDA) approved chenodiol (Ctexli, Mirum Pharmaceuticals Inc.) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Chenodiol is the first FDA-approved drug to treat CTX, a very rare lipid storage disease. CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1 resulting in a […]