Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease

By Yahoo! Finance | Sep 20, 2024 at 01:27 PM EST

Miplyffa, in combination with Johnson & Johnson's enzyme inhibitor Zavesca, is approved by the FDA to treat neurological symptoms in adults and children with NPC, a rare genetic disease. This marks a significant advancement in treating this condition.

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