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[DOI] [PubMed] [Google Scholar] 56.Gasbjerg LS, Helsted MM, Hartmann B, Jensen MH, Gabe MBN, Sparre-Ulrich AH, Veedfald S, Stensen S, Lanng AR, Bergmann NC, et al
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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
