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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

What to Ask Before You Get a Shot Without getting into do-it-yourself protocols, its reasonable to ask your clinician: Is this an FDA-approved product or a compounded mixture

Ensuring proper documentation helps prevent claim delays and denials

Patients should seek urgent medical review if neurological symptoms worsen or fail to improve after 46 weeks of appropriate treatment

Without third-party testing, there is no way to know