Zevra Announces Publication of MIPLYFFA® Mechanism of Action Manuscript in Molecular Genetics and Metabolism [Yahoo! Finance]
Zevra Therapeutics, Inc. (ZVRA)
Company Research
Source: Yahoo! Finance
focused on providing therapies for people living with rare disease, today 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 (NPC) 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.” The publication presents data demonstrating that arimoclomol enters the cell and increases the translocation of translation factors EB and E3 (FTEB & TFE3) from the cytosol to the nucleus, a key initial step for triggering a cascade of downstream
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News
- Is Zevra Therapeutics Attractively Priced After Recent Rare Disease Pipeline Progress? [Yahoo! Finance]Yahoo! Finance
- Is Zevra Therapeutics (ZVRA) Undervalued After Its Recent Share Price Pullback? [Yahoo! Finance]Yahoo! Finance
- Zevra Therapeutics Announces Inducement Grants under Nasdaq Listing Rule 5635(c)(4)GlobeNewswire
- Zevra Therapeutics, Inc. Appoints Alicia Secor to Board of Directors; Announces Retirement of Wendy Dixon, PhDGlobeNewswire
- Zevra Therapeutics, Inc. Announces CFO Transition [Yahoo! Finance]Yahoo! Finance
ZVRA
Earnings
- 11/5/25 - Beat
ZVRA
Sec Filings
- 12/17/25 - Form 8-K
- 12/9/25 - Form 4
- 12/9/25 - Form 3
- ZVRA's page on the SEC website