Zevra Therapeutics

We’re excited to share that the sponsored genetic testing program developed in partnership with GeneDx, is officially live! NPC Detect expands access to exome sequencing for eligible individuals with suspected Niemann-Pick disease type C (NPC). The goal is to help clinicians reach answers faster, support earlier diagnosis, and improve clinical decision-making in this rare, progressive condition. We’re excited to share that the sponsored genetic testing program developed in partnership with GeneDx, is officially live! NPC Detect expands access to exome sequencing for eligible individuals with suspected Niemann-Pick disease type C (NPC). The goal is to help clinicians reach answers faster, support earlier diagnosis, and improve clinical decision-making in this rare, progressive condition. 

Our President and CEO Neil McFarlane will share the latest company updates in a fireside chat at the H.C. Wainwright BioConnect Investor Conference on Tuesday, May 19, 2026 at 9:00 am ET.   Register for the webcast presentation here: https://lnkd.in/gts_zHNU

Excited to share that we will be presenting two encore posters at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on Monday, May 18, 2026, during the evening poster session. We will be featuring two posters highlighting recent findings in Niemann-Pick type C (NPC) research: 📌 Poster #096 (7:00–8:00 PM) “Efficacy of arimoclomol combined with miglustat at months 3, 6, 9, and 12 of the double-blind, randomized, placebo-controlled NPC002 trial” 📌 Poster #129 (8:00–9:00 PM) “Analysis of NPC1 genotypes: findings from the US arimoclomol expanded access program for Niemann-Pick type C” #SIMD2026 #RareDisease #NiemannPickTypeC #NPC 

Zevra is wearing red for #REDS4VEDS Today, we stand with the Vascular Ehlers-Danlos Syndrome (VEDS) community, a group facing a rare, life-threatening genetic condition that affects connective tissue, including blood vessels and organs. At Zevra, we believe awareness is a powerful first step toward earlier diagnosis, better care, and meaningful progress in research. Learn more about VEDS: https://lnkd.in/eN-EKiBa

Next week, the Zevra team will be dividing and conquering as we head to two key scientific meetings focused on advancing care and innovation in rare disease. We’re excited to connect with clinicians, researchers, and partners at: 📍American Psychiatric Association (APA) Annual Meeting 2026 - Booth #1837 📍Society for Inherited Metabolic Disorders (SIMD) Annual Meeting 2026 - Booths #15 & #36 If you’re attending either meeting, we welcome the opportunity to connect and share updates, insights, and progress across our pipeline. See you there.

At Zevra, our mission is clear: we redefine what is possible in bringing life-changing therapies to people living with rare diseases. Our values guide us every day, shaping how we work, collaborate, and stay focused on what matters most – patients and families. #RareDisease #CompanyCulture

Zevra Therapeutics reports first quarter 2026 financial results and provides a corporate update.   Join our LIVE webcast TODAY at 4:30 p.m. ET: https://lnkd.in/eS9MmUzz   For more, find the full release here:  https://lnkd.in/eSaz9KHi   #RareDiseases #InvestorRelations #IR 

We’re excited to share that Zevra Therapeutics was featured in PharmaBoardroom’s recent rare disease addition. The feature brings together key voices across the U.S. Food and Drug Administration, National Institutes of Health, policy and advocacy leaders, leading research institutions, and industry executives, all focused on advancing progress for the rare disease community. We’re honored to be included among stakeholders working to drive innovation, collaboration, and meaningful impact for patients and families. Read the full article here: https://lnkd.in/evY5nJ4J #RareDisease #HealthcareLeadership 

That’s a wrap on #AANAM! Our team enjoyed connecting with the neurology community and engaging with attendees at our booth. Thanks to everyone who stopped by. We’re excited by the momentum and look forward to what’s next.

Zevra Therapeutics will report first quarter 2026 financial results and provide a corporate update on Wednesday, May 6, 2026, with a live webcast at 4:30 p.m. ET. For registration details, see the release: https://lnkd.in/e4QeJrZk #RareDiseases #InvestorRelations #IR