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 looking forward to attending the Ara Parseghian Medical Research Foundation Annual Conference 2026.This important ...
05/29/2026

We’re looking forward to attending the Ara Parseghian Medical Research Foundation Annual Conference 2026.

This important meeting brings together researchers, clinicians, and advocates dedicated to advancing understanding and care for Niemann-Pick disease type C (NPC). We’re excited to connect with the community and continue supporting progress for patients and families.

What a meaningful week spent engaging across two impactful scientific meetings: American Psychiatric Association Annual ...
05/22/2026

What a meaningful week spent engaging across two impactful scientific meetings: American Psychiatric Association Annual Meeting 2026 and Society for Inherited Metabolic Disorders Annual Meeting 2026.

From scientific exchange to thoughtful conversations with clinicians, researchers, and partners, both meetings offered valuable perspectives on the evolving landscape of rare disease. We’re grateful for the opportunity to share updates, learn from the community, and explore new ways to advance care for patients and families.

Thank you to everyone who connected with the Zevra team!

We’re excited to share that the sponsored genetic testing program developed in partnership with GeneDx, is officially li...
05/19/2026

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.

Excited to share that we will be presenting two encore posters at the Society for Inherited Metabolic Disorders (SIMD) A...
05/18/2026

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”

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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/

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Celebration, FL

Opening Hours

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

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+18889581253

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