FDA Approves New Drug to Treat Niemann-Pick Disease, Type C

SILVER SPRING, Md., Sept. 24, 2024 /PRNewswire/ — Today, the U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms….