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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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Regulatory note: Ara-290 is not approved by the FDA or any regulator it is investigational and has only reached Phase 2

Clinicians may recommend smaller meals, softer foods, thorough chewing, lower-fat choices, or temporary liquid nutrition when symptoms become significant