Publication of Positive MIPLYFFA Pediatric Substudy Data in Infants with Niemann-Pick Disease Type C – Zevra Therapeutics
Zevra Therapeutics announced the publication of “Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy” in Molecular Genetics and Metabolism Reports…. read more.




