Zevra Therapeutics (NASDAQGS: ZVRA), a commercial-stage company focused on therapies for rare diseases, announced that four posters on MIPLYFFA (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), held Sept. 2-6, 2025, in Kyoto, Japan. MIPLYFFA, approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC), will be highlighted in a Best Poster award-winning presentation (#BP-19) detailing its unique mechanism of action targeting NPC pathophysiology. Additional presentations will feature positive new data from a pediatric substudy in patients younger than two years old and a prespecified efficacy analysis in patients on miglustat who switched from placebo to MIPLYFFA.
To view the full press release, visit https://ibn.fm/VMls7
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage company combining science, data and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
For more information about the company, please visit www.zevra.com
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