Zevra Therapeutics

12/10/2025

One year ago, we took a pivotal step forward in our mission and today we celebrate a true lifeline for patients living with Niemann‑Pick Type C (NPC).

Meet Cole Stites. At age 14 his world flipped when he experienced a grand-mal seizure and was diagnosed with NPC, a rare, devastating disease that robs children of their ability to walk, speak and live fully.

Today, Cole is back throwing his first pitch of the season and living life with renewed hope.

As we mark this anniversary, we honor every patient and family who trusted us. Their courage fuels our commitment to continue advancing transformational therapies for rare diseases.

Here’s to another year of making a difference!

Niemann-Pick Type C, or NPC is a relentlessly progressive and fatal genetic disorder that robs children of their ability to speak, think clearly, swallow, walk, and move. And now there’s a new FDA-approved medication that could be a game changer for these kids

11/27/2025

This Thanksgiving, we’re grateful for the patients, families, caregivers, advocates, and researchers who inspire our mission every day.

In the rare disease community, progress happens through collaboration, courage, and hope. We’re thankful to be part of a collective effort working toward brighter futures for all those affected by rare diseases.

From all of us at Zevra Therapeutics, we wish you a safe and joyful Thanksgiving filled with connection, reflection, and gratitude.

10/30/2025

As Niemann-Pick disease Awareness Month comes to a close, we honor the courage of teens like Connor Martin, whose journey with Niemann-Pick disease type C (NPC) reminds us that every moment matters.

Connor and his family never stopped searching for answers. Today, thanks to advances in research and treatment, they’re finding more time, memories, and hope.

Read Connor’s inspiring story via CBS Minnesota: https://bit.ly/4hvo3TG

When a 17-year-old Minnesota boy got sick with a mysterious illness, his mom went on a quest for answers.

10/29/2025

10/20/2025

Zevra Therapeuticsherapeutics is proud to partner with the National Urea Cycle Disorders Foundation as the sole sponsor of Check Ammonia Month.

Together, we’re raising awareness of the lifesaving importance of early ammonia testing to help identify individuals with urea cycle disorders (UCDs) who may still be undiagnosed. For those living with UCDs, a quick ammonia level check can mean the difference between life and death.

Learn how to check ammonia here: https://bit.ly/3BOeoXI

10/19/2025

On Global Niemann-Pick Disease Awareness Day, we honor the strength and resilience of the community.

At Zevra Therapeutics, we remain dedicated to expanding global access to our foundational NPC treatment, with the goal of bringing hope and meaningful impact to patients and their loved ones.

10/16/2025

The DiSCOVER Trial is an opportunity to be part of a scientific process designed to evaluate the safety and effectiveness of celiprolol in an effort to bring a much-needed treatment option for people living with VEDS in the U.S.

Learn more here: https://bit.ly/3VDCFGu

10/14/2025

October is Niemann-Pick Disease Awareness Month, a time to honor patients and families facing this devastating rare disease and to strengthen awareness.

At Zevra, we are committed to changing what’s possible for those living with Niemann-Pick disease type C (NPC). Our foundational therapy is the only treatment shown to directly address the underlying pathology of NPC, with long-term, meaningful clinical outcomes and sustained efficacy and safety demonstrated over 5 years.

We continue to be inspired by the resilience of the NPC community and remain dedicated to expanding global access to our foundational treatment to bring hope and impact to patients and their loved ones.

10/10/2025

October marks VEDS Action Month, a time to raise awareness for Vascular Ehlers-Danlos Syndrome (VEDS), a rare, life-threatening genetic condition that often goes underdiagnosed and undertreated.

At Zevra Therapeutics, we stand with patients, families, and advocates driving awareness, education, and support.

Their strength underscores the urgent need for new treatment options and inspires our work to advance celiprolol as a potential treatment option for those living with VEDS.

Together, we can shine a light on VEDS, strengthen the community’s voice, and continue moving research forward.

10/09/2025

Our team was honored to connect with the VEDS community at the Society for Vascular Medicine (SVM) 2025 Vascular Scientific Sessions.

As proud sponsors of this year’s meeting, we value the opportunity to engage with leaders advancing vascular medicine and to stand alongside patients, caregivers, and advocates during VEDS Action Month.

Together, we share a mission of driving progress in care and developing new potential treatment options for VEDS.

We’re grateful to the SVM and VEDS community for such an inspiring meeting.

09/30/2025

It was a privilege to be part of The Ehlers-Danlos Society 2025 International Scientific Symposium, an incredible event where the global EDS and HSD community came together to share knowledge and hope for the future.

We are deeply grateful to the community for inspiring the advancement of celiprolol in an effort to bring a much-needed treatment option for people living with in the U.S.

09/22/2025

The DiSCOVER Trial is designed to make it as convenient and easy as possible to participate. Start by finding out if you meet the requirements to enroll in the trial. Just complete the short, online questionnaire.

Learn more here: https://bit.ly/3VDCFGu