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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

The mechanism behind this is synergistic rather than additive

HIF-1, as a pivotal regulator response to oxygen homeostasis, metabolism, inflammation and in crosstalk with mitochondria, is the underlying mechanism of determining disease progression in PH [36,37,38,39]