Zevra Therapeutics

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/

01/28/2026

Zevra Therapeutics is heading to the 22nd Annual WORLDSymposium.

We’re excited to share new data and insights through a podium presentation, poster sessions, and a satellite symposium during the meeting.

Read more in our press release here: https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-present-multiple-analyses-miplyffar-22nd

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.