FDA Approves First Treatment for Niemann-Pick Disease, Type C

SILVER SPRING, Md., Sept. 20, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat...