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September 20 Product: Zevra Therapeutics’ Miplyffa Indication: Niemann-Pick disease type C Patients with Niemann-Pick disease type C (NPC) have their first treatment after the FDA approved Zevra Therapeutics’ Miplyffa for the ultra-rare, progressive neurodegenerative disease

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10.1126/science.1060077 73 WangK.SachdevaA.CoxD