Zevra Therapeutics

Zevra Therapeutics We are a purpose-driven company focused on bringing life-changing therapeutics to people living with rare diseases.

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.

We’re excited to share that Zevra Therapeutics was featured in PharmaBoardroom’s recent rare disease addition. The featu...
04/28/2026

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://pharmaboardroom.com/articles/spearheading-the-charge-against-rare-disease/

Good luck to everyone running the 130th Boston Marathon today! Our team is cheering on all the runners, volunteers, and ...
04/20/2026

Good luck to everyone running the 130th Boston Marathon today!

Our team is cheering on all the runners, volunteers, and supporters who make this incredible day possible. Wishing you a strong race and great weather out there!

We’re excited to attend the 2026 AAN Annual Meeting, April 18–22 in Chicago, IL. If you’re attending  , we invite you to...
04/16/2026

We’re excited to attend the 2026 AAN Annual Meeting, April 18–22 in Chicago, IL.

If you’re attending , we invite you to stop by booth #4834 to connect and learn more about our ongoing efforts focused on Niemann-Pick disease type C (NPC).

We look forward to engaging with clinicians, researchers, and advocates working to advance care and understanding of rare neurological diseases.

Niemann-Pick disease type C (NPC) is a rare, progressive genetic disorder that affects patients and families every day.U...
04/07/2026

Niemann-Pick disease type C (NPC) is a rare, progressive genetic disorder that affects patients and families every day.

Understanding NPC starts with learning more and every bit of awareness helps support those living with it.

Visit https://www.learnnpc.com to learn more about NPC and how you can help raise awareness.

April Fools’ may be a day for jokes, but for the rare disease community, every day is serious.At Zevra, we are committed...
04/01/2026

April Fools’ may be a day for jokes, but for the rare disease community, every day is serious.

At Zevra, we are committed to bringing life-changing therapies to people living with rare diseases. We honor the patients, families, and caregivers who face these challenges every day, and we are dedicated to raising awareness, advancing research, and delivering on our mission.

Together, we work toward a future where rare disease is met with solutions, not compromise.

For families living with VEDS, the DiSCOVER Trial represents hope for the future. Each participant helps accelerate rare...
03/26/2026

For families living with VEDS, the DiSCOVER Trial represents hope for the future. Each participant helps accelerate rare disease science and help shape what could be next in vascular care.

If you or someone you know meets the criteria, learn more about participation details here: https://discoverceliprolol.com/

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

We’re pleased to announce our partnership with GeneDx to launch a Sponsored Genetic Testing Program for patients in the ...
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

We’re excited to welcome Justin Renz as our new Chief Financial Officer. With 25+ years of biopharma finance leadership ...
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

Last night our President and CEO Neil McFarlane, attended the inaugural CNBC Cures Summit hosted by CNBC, a powerful gat...
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.

Today is Rare Disease Day, a moment to recognize the millions of people worldwide living with rare conditions, including...
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

Last week, the Zevra team had the honor of attending the Firefly Dreams Gala, a powerful evening dedicated to supporting...
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.

Address

Celebration, FL

Opening Hours

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

Telephone

+18889581253

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