FDA Approves CTEXLI, the First-Ever Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disorder Mirum Pharmaceuticals, Inc. announced that the FDA has approved CTEXLI (chenodiol) tablets as the first and only treatment for cerebrotendinous xanthomatosis (CTX), a rare, progressive, and debilitating metabolic disease. CTX is caused by a deficiency of chenodeoxycholic acid (CDCA), leading to … Continue reading CTEXLI Approved for Cerebrotendinous Xanthomatosis; SIGX1094 Wins Fast Track for Diffuse Gastric Cancer; Bavarian Nordic’s Chikungunya Vaccine Cleared for Ages 12+; UX111 Gene Therapy Gains Priority Review for Sanfilippo Syndrome; SUBLOCADE Label Update Approved