#USA #Rare Diseases #Norwell, Massachusetts #NORD #Rare Impact Awards

Celebrating the 2026 NORD Rare Impact Award Honorees

On September 24, 2026, the National Organization for Rare Disorders (NORD) proudly announced the honorees of the 2026 Rare Impact Awards. This prestigious recognition is dedicated to individuals and organizations that are significantly contributing to the advancement of research, innovation, and advocacy in the field of rare diseases. This year, the honorees include biopharma leaders, medical scientists, and passionate advocates united in their mission to improve the lives of over 30 million Americans afflicted by rare diseases.

The Importance of the NORD Rare Impact Awards

NORD's Rare Impact Awards are an essential initiative that highlights the significant challenges faced by those living with rare disorders. Although there are over 10,000 known rare diseases, a staggering 95% currently lack approved treatments, leading the organization to emphasize the urgent need for scientific progress. According to Pamela K. Gavin, NORD’s Chief Executive Officer, “NORD is proud to honor these companies and organizations for pioneering new therapies and advancing research that can change what is possible for individuals and families affected by these rare diseases.”

Recognizing Industry Innovators

This year's honorees include groundbreaking companies at the forefront of developing therapies that have markedly improved patient outcomes:

• - Mighty Therapeutics for FORZINITY™, the first treatment targeting Barth syndrome, aimed at enhancing muscle strength and overall quality of life.
• - UCB for KYGEVVI, the sole FDA-approved treatment for pediatric and adult patients with thymidine kinase 2 deficiency.
• - Fondazione Telethon for introducing Waskyra™, a pioneering gene therapy approved for treating Wiskott-Aldrich syndrome via a nonprofit organization.
• - Omeros, for YARTEMLEA®, designed for patients experiencing complications post-hematopoietic stem cell transplants.
• - Jazz Pharmaceuticals for MODEYSO™, targeting aggressive brain tumors in young patients.
• - Verastem Oncology for AVMAPKI® FAKZYNJA® CO-PACK, an important treatment for recurrent low-grade serous ovarian cancer.

Championing Advocates and Organizations

The 2026 awards recognize not only corporate achievers but also dedicated individuals and organizations whose advocacy efforts elevate awareness and improve the lives of those affected by rare diseases:

• - Foundation for Prader-Willi Research (FPWR) was honored with the Abbey S. Meyers Leadership Award for its impactful work in patient-centered research.
• - Dr. Stephanie E. Haridopolos received the Policy Changemaker Award for her significant role in adding critical conditions to newborn screening panels.
• - Dr. Julieta Bonvin Sallago, Lily Emmanuel, and Dr. Maureen Helgren were awarded Community Champion Awards for their transformative work in their respective areas, focusing on educating and advocating within diverse communities.
• - Mark Skinner was recognized with a Lifetime Achievement Award for his groundbreaking advocacy for bleeding disorders.
• - Young leader Paridhi Tyagi was acknowledged for her initiatives aimed at raising awareness and inclusivity concerning genetic diseases among youth.

Inspirational Contributions for All

Gavin reaffirmed the intrinsic power of individuals from various sectors to bring about change, stating, “Each of these 2026 Rare Impact Award winners demonstrates the power we have as individuals to drive change in our communities.” The recognition ceremony for Community Champions, Policy Changemakers, and Leadership Award winners will occur at the NORD Breakthrough Summit on October 26-27 in Washington, D.C. Meanwhile, the Scientific and Medical Trailblazers were earlier honored at a dedicated symposium.

About NORD

Founded in 1983, NORD remains the leading independent nonprofit organization dedicated to enhancing the health and lives of the millions of Americans grappling with rare diseases. Through collaboration with more than 360 disease-specific patient organizations and numerous centers of excellence nationwide, NORD aims to further research efforts, care, and legislative policy improvements in this field. To discover more about the pioneering efforts of NORD and the honorable recipients of the 2026 Rare Impact Awards, please visit rarediseases.org.