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Zevra Therapeutics

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Celebration, FL
Zevra Therapeutics

We are a nimble rare disease therapeutics company bringing new therapies to patients.

We are a nimble rare disease therapeutics company comprised of expert scientists, patient advocates, development strategists, commercialization specialists, and business development gurus with a proven record of bringing new therapies to patients.

03/11/2026

We’re excited to attend the 2026 ACMG Annual Clinical Genetics Meeting, March 10–14 in Baltimore, MD.

Our team will present a poster highlighting the various NPC1 genotypes in Niemann-Pick disease type C patients on Friday, March 13, at 10:30 a.m. ET and host a booth throughout the meeting.

We look forward to engaging with the genetics community and sharing our latest work.

If you’re attending , we invite you to stop by our booth #2008 to connect!

03/10/2026

We’re pleased to announce our partnership with GeneDx to launch a Sponsored Genetic Testing Program for patients in the U.S. with suspected Niemann-Pick disease type C (NPC).

By expanding access to free exome sequencing to qualified patients, we aim to help clinicians confirm diagnoses sooner and support more informed care decisions for the NPC community.

This partnership underscores our commitment to people living with rare diseases, reducing uncertainty for families and helping patients access appropriate care as quickly as possible. Read the press release: https://bit.ly/3NqH3Z4

03/05/2026

We’re excited to welcome Justin Renz as our new Chief Financial Officer.

With 25+ years of biopharma finance leadership spanning capital markets, strategic transactions, and commercial-stage operations, Justin brings strong experience and operational insight. His leadership will help advance our mission of bringing life-changing therapies to people living with rare diseases.

Please join us in welcoming Justin to Zevra. Read the release: https://bit.ly/472pHbN

03/04/2026

Last night our President and CEO Neil McFarlane, attended the inaugural CNBC Cures Summit hosted by CNBC, a powerful gathering of leaders focused on accelerating breakthroughs in rare disease research.

The summit’s call to reimagine healthcare and remove barriers to innovation strongly aligns with Zevra Therapeutics’ mission and commitment to the rare disease community.

We’re encouraged by the momentum and collaboration driving meaningful change for patients and families.

02/28/2026

Today is Rare Disease Day, a moment to recognize the millions of people worldwide living with rare conditions, including Niemann-Pick disease type C.

At Zevra, raising awareness for rare diseases — and particularly NPC — is not limited to one day or one month. Our commitment to education, understanding, and the rare disease community continues all year long.

Learn more about how you can get involved and help raise awareness beyond Rare Disease Day at https://www.learnnpc.com/npc-symptoms/ -support

02/27/2026

Last week, the Zevra team had the honor of attending the Firefly Dreams Gala, a powerful evening dedicated to supporting rare disease research and education, specifically Niemann-Pick disease type C (NPC).

This year’s cowboy chic theme brought energy and heart to an unforgettable night. Most importantly, it was a moving reminder of why raising awareness for rare diseases like NPC matters so deeply.

We’re proud to stand alongside this community and support the mission.

02/25/2026

Did you know?

Niemann-Pick disease type C is inherited in an autosomal recessive pattern, meaning both parents must carry a copy of the affected gene for a child to develop the condition.

While NPC is rare, understanding its genetic nature and clinical presentation can support earlier testing, diagnosis, and access to appropriate resources and care.

Learn more about NPC and the path to diagnosis at https://www.learnnpc.com/diagnosis-and-testing/

02/18/2026

Did you know?

NPC symptoms can vary widely from person to person and may include liver or spleen enlargement, developmental delays, difficulty with balance and coordination, eye movement abnormalities, and cognitive challenges.

Because these symptoms can overlap with other conditions, individuals with NPC may experience delays in diagnosis. Increased disease awareness can help families and healthcare providers recognize the signs sooner.

Explore common symptoms of NPC at https://www.learnnpc.com/npc-symptoms/

02/11/2026

Did you know?

Niemann-Pick disease type C (NPC) is a rare, inherited lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes. These mutations lead to the buildup of lipids in cells, resulting in progressive dysfunction of the brain and other organs.

NPC can affect people of all ages, and its variability often makes diagnosis challenging. Raising awareness is an important step toward earlier identification.

Learn more about NPC at https://www.learnnpc.com/

02/06/2026

Wrapping up an action-packed week at and feeling incredibly grateful.

It’s always deeply meaningful to spend time with the rare disease community, connecting with patients, caregivers, advocates, clinicians, and innovators who are united by a shared commitment. The conversations and collaborations throughout the week reinforced the power of coming together.

We’re especially thankful to everyone who stopped by the Zevra booths and to those who attended our presentations or symposium. Your engagement and insights made the week even more impactful. Thank you to the organizers for another outstanding event. We look forward to carrying this momentum forward.

02/02/2026

Attending the 22nd Annual WORLDSymposium? Stop by and see us in person.

Visit Zevra at booths #213 and #507 during WORLDSymposium, taking place February 2–6, 2026, in San Diego, CA. Our team looks forward to connecting with attendees to discuss our ongoing work in rare diseases, including Niemann-Pick disease type C.

02/01/2026

February is Rare Disease Awareness Month, and we’re proud to stand with individuals and families impacted by rare diseases — including Niemann-Pick disease type C (NPC).

At Zevra, our commitment goes beyond awareness. We are dedicated to supporting the rare disease community every day of the year.

This month, we’re shining a spotlight on NPC — a progressive, rare genetic disease that can be difficult to diagnose due to its wide range of symptoms. Increasing awareness can help support earlier recognition and treatment.

Learn more at: https://zevra.com/niemann-pick-disease-type-c/

Address

Celebration, FL

Opening Hours

Monday	9am - 5pm
Tuesday	9am - 5pm
Wednesday	9am - 5pm
Thursday	9am - 5pm
Friday	9am - 5pm

Telephone

+18889581253

Website

http://zevra.com/

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