Zevra Therapeutics, Inc. (NASDAQ:ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, 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. This multicenter, open-label pediatric substudy evaluated arimoclomol, in addition to concomitant miglustat, in five children with Niemann-Pick disease type C (NPC) aged 12 to <24 months at enrollment, assessing safety, tolerability, and pharmacokinetics over up to 36 months of treatment.
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