Zevra Therapeutics presents real-world data on Miplyffa

Zevra Therapeutics (ZVRA) announced the presentation of four posters highlighting positive new data on Miplyffa for the treatment of Niemann-Pick Disease Type C at the 22nd Annual WORLDSymposium. “These data highlight MIPLYFFA’s potential to meaningfully stabilize disease progression across a broad spectrum of NPC patients, including adults who have historically had limited clinical data,” said Adrian Quartel, M.D., FFPM, Zevra’s Chief Medical Officer. “As controlled clinical trial results are reinforced by long-term real-world experience, we continue to strengthen our understanding of Miplyffa’s impact and advance our mission to deliver meaningful therapies for people living with rare diseases.”