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…

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