Zevra Therapeutics (ZVRA) announces the publication of “Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease” in Molecular Genetics and Metabolism. “This elucidation of MIPLYFFA’s highly differentiated mechanism of action marks a critical step in understanding its interactions with Niemann-Pick disease type C at a cellular level,” said Adrian Quartel, MD, FFPM, Zevra’s Chief Medical Officer. “These insights further confirm that MIPLYFFA addresses the underlying pathology of NPC and supports the long-term treatment benefit observed in our clinical trials. Our findings may inform more effective treatment strategies for patients.”
Elevate Your Investing Strategy:
- Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.
- Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Read More on ZVRA:
- Zevra Therapeutics closes sale of Rare Disease PRV for $150M
- Zevra Therapeutics management to meet with Cantor Fitzgerald
- Optimistic Buy Rating for Zevra Therapeutics Driven by Miplyffa Launch and Strategic Financial Moves
- Zevra Therapeutics price target raised to $22 from $20 at Guggenheim
- Zevra Therapeutics: Promising Outlook with Strong Financials and Strategic Focus on Rare Diseases