New Real-World Data Show MIPLYFFA's Potential to Stabilize NPC Disease Progression

Zevra Therapeutics presented four posters highlighting positive real-world data on MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium. The data showed that MIPLYFFA stabilized disease progression in a broad spectrum of NPC patients, including adults with limited clinical data. The real-world evidence demonstrated durable treatment effects and sustained clinical benefit with continued use over time.