Genetic and Congenital

FDA Approves First Therapy for Children with Neurofibromatosis Type 1

The U.S. Food and Drug Administration has approved selumetinib (Koselugo) for the treatment of pediatric patients, ≥2 years of age, with neurofibromatosis type 1 (NF1). This is the first drug approved by the FDA to treat NF1. NF1 is a genetic disorder of the nervous system that causes tumors to...