The FDA has approved Miplyffa, the first-ever treatment for Niemann-Pick disease, type C (NPC). Miplyffa, used with the enzyme inhibitor miglustat, is approved to treat neurological symptoms in adults and children aged 2 and older. NPC is a rare genetic disorder that affects the bodys ability to process cholesterol and lipids, leading to severe neurological complications. This approval offers new hope for patients affected by this debilitating condition.