Zevra Therapeutics

09/02/2026

At Zevra Therapeutics, our mission begins with our people.
Whether advancing clinical research, supporting patients, or bringing innovative therapies to market, we're united by a common purpose: making a meaningful difference for patients and families.

08/29/2026

Thank you to the SSIEM attendees for an engaging and impactful meeting focused on advancing care for people living with rare metabolic diseases.

We appreciate the opportunity to connect with clinicians, researchers, advocates and the broader rare disease community working to improve outcomes for people living with Niemann-Pick disease type C (NPC).

08/25/2026

We are thrilled to be presenting two posters featuring new real-world data on arimoclomol in Niemann-Pick disease type C (NPC) at SSIEM 2026.

The presentations highlight findings from U.S. and German early access programs and will be presented during the dedicated poster walk on Wednesday, August 26, from 5:30–6:30 p.m. CEST.

Read the press release: https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-present-new-long-term-real-world-data

Visit us at booth S12 in exhibition hall 5 to learn more!

08/22/2026

We’re heading to SSIEM 2026!

Zevra will be presenting new real-world data on arimoclomol in people living with Niemann-Pick disease type C (NPC) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, August 25-28.

Visit us at booth S12 in exhibition hall 5 to learn more!

08/17/2026

Living with a rare disease often means navigating a long and uncertain diagnostic journey.

Niemann-Pick disease type C (NPC) is a rare, progressive genetic disorder that can affect children and adults, often presenting with symptoms that may be mistaken for other conditions.
Increasing awareness can help shorten the path to diagnosis and connect families with critical resources and support.

Learn more about the signs, symptoms, and patient resources available at LearnNPC.com.

08/12/2026

Honored that Zevra has been nominated for the 2026 Prix Galien USA Awards in the Best Product for Rare/Orphan Diseases category. This recognition reflects our commitment to advancing meaningful therapies for people living with rare diseases.

𝗣𝗿𝗶𝘅 𝗚𝗮𝗹𝗶𝗲𝗻 𝟮𝟬𝟮𝟲 𝗻𝗼𝗺𝗶𝗻𝗲𝗲𝘀 𝗼𝗳𝗳𝗶𝗰𝗶𝗮𝗹 𝘀𝗲𝗹𝗲𝗰𝘁𝗶𝗼𝗻 𝗮𝗻𝗻𝗼𝘂𝗻𝗰𝗲𝗱!
The 2026 USA nominees for "Best Biotechnology Product," "Best Pharmaceutical Product" and "Best Product for Rare/Orphan Diseases" have been announced.
Read more: https://prn.to/4gqUFiw

08/03/2026

We’re pleased to announce the publication of “Long-term Real-World Safety and Effectiveness of Arimoclomol in Individuals with NPC: Outcomes from the U.S. Early Access Program Over a 4-Year Period” in Molecular Genetics and Metabolism.

This publication highlights long-term real-world outcomes from the U.S. Early Access Program and contributes to the growing body of evidence supporting arimoclomol in NPC.

Read our press release to learn more: https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-announces-publication-four-year-real-world

07/25/2026

Today, on Genetic Testing Action Day, we recognize the important role genetic testing can play in helping people with rare diseases get answers sooner.

For families affected by Niemann-Pick disease type C (NPC) and other rare genetic conditions, an accurate diagnosis can be a critical first step toward care and support.

At Zevra, we're committed to raising awareness of rare diseases and working to help shorten the diagnostic journey.

07/22/2026

Zevra Therapeutics will report second quarter 2026 financial results and provide a corporate update on Wednesday, August 5, 2026, with a live webcast at 4:30 p.m. ET.

For registration details, see the release: https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-announces-details-q2-2026-financial-results

07/16/2026

July is Disability Pride Month.

Many individuals living with Niemann-Pick disease type C (NPC) experience disabilities that impact daily life. This month, we recognize the strength, resilience, and advocacy of the NPC community and reaffirm the importance of inclusion, accessibility, and amplifying patient voices.

At Zevra, we are grateful to the patients, families, caregivers, and advocates who share their experiences and help advance understanding of NPC every day.