Press release
Zevra Therapeutics Announces Upcoming Oral Presentation at the International Niemann-Pick Disease Alliance (INPDA) Meeting and Poster Presentations at the Child Neurology Society (CNS) Conference on MIPLYFFA® (arimoclomol) and OLPRUVA® (sodium phenylb...
Zevra Therapeutics Announces Upcoming Oral Presentation at the International Niemann-Pick Disease Alliance (INPDA) Meeting and Poster Presentations at the
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Zevra Therapeutics Announces Upcoming Oral Presentation at the International Niemann-Pick Disease Alliance (INPDA) Meeting and Poster Presentations at the Child Neurology Society (CNS) Conference on MIPLYFFA® (arimoclomol) and OLPRUVA® (sodium phenylbutyrate) \n MIPLYFFA, the only therapy for Niemann-Pick disease type C (NPC) shown to stop disease progression at 12 months, has been administered to more than 270 patients, representing the largest data set of NPC clinical trial patients OLPRUVA offers the established efficacy and safety of sodium phenylbutyrate in a novel and convenient formulation for the treatment of certain urea cycle disorders CELEBRATION, Fla., Sept. 18, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced an upcoming oral presentation at the International Niemann-Pick Disease Alliance (INPDA) Face-to-Face Meeting and several poster presentations at the Child Neurology Society (CNS) Annual Meeting, which will be reviewing clinical data on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) for the treatment of Niemann-Pick disease type C (NPC) and OLPRUVA® (sodium phenylbutyrate) for the treatment of certain patients living with urea cycle disorders (UCDs). INPDA Face-to-Face Meeting - September 18-21, 2025 in Puerto Iguazú, Misiones, Argentina Title:Changing the course of NPC: long-term evidence for disease modification in a heterogenous populationDate:Saturday, September 20, 2025Presenter:Eugen Mengel, M.D.; CEO of SphinCS and Principal Investigator for MIPLYFFA Phase 3 CNS Annual Meeting - October 8-11, 2025 in Charlotte, North Carolina Title:Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to","length":5086,"tagName":"div"}]