GREENWICH, Conn., September 30, 2024 – Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” is proud to announce that its 7th annual DART to the Finish Charity Walk, held on September 28, …
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FDA Approves 2nd New Drug to Treat NPC
FDA Approves New Drug to Treat Niemann-Pick Disease, Type C | FDA For Immediate Release:September 24, 2024 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. “This is the second treatment …
FDA Approves 1st Treatment for NPC
FDA Approves First Treatment for Niemann-Pick Disease, Type C | FDA For Immediate Release:September 20, 2024 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 neurological symptoms associated with NPC in …