Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)
Data published in Molecular Genetics and Metabolism Reports suggest MIPLYFFA (arimoclomol) was well tolerated in pediatric patients with no new safety signals observed Data published in Molecular Genetics and Metabolism Reports suggest MIPLYFFA (arimoclomol) was well tolerated in pediatric patients with no new safety signals observed