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
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 phenylbutyr
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