US Approves Treatment for Niemann-Pick Disease-Type C
The US Food and Drug Administration (FDA) approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved […]