NORWELL, Mass., Sept. 24, 2026 /PRNewswire/ — The National Organization for Rare Disorders (NORD®) is honoring a distinguished group of biopharma industry leaders, medical scientists, and patient advocates for their contributions to improving the lives of the more than 30 million Americans living with rare diseases through the annual NORD Rare Impact Awards®.

The 2026 honorees include innovative companies whose treatments have significantly improved rare disease patient outcomes, trailblazing scientists in rare disease research, and passionate advocates raising awareness and spearheading legislative change.

Industry Innovators in Rare Disease

Among this year’s honorees are five companies and one nonprofit who — alongside impacted patient communities — celebrated recent U.S. Food and Drug Administration (FDA) approvals for first-of-their-kind rare disease therapies.

“With approximately 95% of more than 10,000 known rare diseases still lacking an approved treatment, advancing scientific progress is critical,” said Pamela K. Gavin, NORD Chief Executive Officer. “NORD is proud to honor these companies and nonprofit organizations for pioneering new therapies and advancing research that can change what is possible for individuals and families affected by these rare diseases. Their progress in addressing the unique challenges of these conditions is also contributing knowledge, approaches, and breakthroughs that help move science and the entire rare disease field forward.”

The 2026 Industry Innovator honorees are:

• Mighty Therapeutics for FORZINITY™, the first therapy for Barth syndrome — a very rare, progressive, and life-limiting genetic disease affecting mitochondria — shown to improve muscle strength and quality of life.
• UCB for KYGEVVI, the first and only FDA-approved treatment for adults and children with thymidine kinase 2 deficiency (TK2d) whose symptoms began at or before 12 years of age.
• Fondazione Telethon for Waskyra™, a breakthrough gene therapy for Wiskott-Aldrich syndrome (WAS) that represents the first FDA-approved gene therapy for a primary immunodeficiency disorder and the first approved gene therapy developed by a nonprofit organization.
• Omeros for YARTEMLEA®, the first and only FDA-approved treatment for adults and children 2 years and older with hematopoietic stem cell transplant–associated thrombotic microangiopathy (TA-TMA), a significant and often fatal complication of stem cell transplantation.
• Jazz Pharmaceuticals for MODEYSO™, the first and only FDA-approved treatment for one of the most aggressive brain tumors impacting children and young adults, recurrent H3K27M-mutant diffuse midline glioma (DMG).
• Verastem Oncology for AVMAPKI^® FAKZYNJA^® CO-PACK, the first treatment specifically approved for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

NORD is also proud to recognize individuals and organizations dedicated to advocating and improving life for the one in 10 Americans living with a rare disorder. This year, those honorees include:

• Foundation for Prader-Willi Research (FPWR), recipient of the Abbey S. Meyers Leadership Award for leading patient-centered research through its Global PWS Registry on NORD’s IAMRARE® platform that contributed to an FDA approved treatment.
• Stephanie E. Haridopolos, MD, DABFM, recipient of the Policy Changemaker Award for facilitating the Department of Health and Human Services’ addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Newborn Screening Panel (RUSP).
• Julieta Bonvin Sallago, MD of Connecticut, recipient of a Community Champion Award for her leadership in translating medical information, improving programming, and expanding recruitment for NORD’s Living Rare Study as a volunteer on behalf of Spanish-speaking communities.
• Lily Emmanuel of Colorado, recipient of a Community Champion Award for her leadership as an advocate and organizer with NORD Running for Rare® in Colorado.
• Maureen Helgren, PhD of Connecticut, recipient of a Community Champion Award for integrating rare disease education into the curriculum at Quinnipiac University’s Frank H. Netter, MD School of Medicine and fostering student advocacy through the University’s NORD Students for Rare® chapter.
• Susan A. Berry, MD of Minnesota, recipient of a Medical & Scientific Trailblazer Award for defining a national model of care for inborn errors of metabolism at the University of Minnesota Medical School NORD Rare Disease Center of Excellence, advising on national policies for newborn screening and medical nutrition, and serving on NORD’s Board of Directors and Scientific & Medical Advisory Committee.
• Stephen Kingsmore, MD, DSc of California, recipient of a Medical & Scientific Trailblazer Award for founding the Rady Children’s Institute for Genomic Medicine NORD Rare Disease Center of Excellence, where he pioneered whole genome sequencing, rapid genetic testing as a clinical tool, and the first comprehensive genomic carrier screening test.
• Mark Skinner, JD of New York, recipient of the Lifetime Achievement Award for his advocacy for bleeding disorders in New York, the U.S., and worldwide through the World Federation of Hemophilia, the National Bleeding Disorders Foundation, the HHS Advisory Committee on Blood and Tissue Safety and Availability, and NORD’s Board of Directors.
• Paridhi Tyagi of New Jersey, recipient of the Youth Leadership Award for founding a genetics club at Millburn High School through NORD Students for Rare® and collaborating with Girl Scouts USA to host three workshops educating Scouts about inclusion, acceptance, and the science behind genetic diseases.

“Each of these 2026 Rare Impact Award winners demonstrates the power we have as individuals to drive change in our communities, whether as an advocate, policymaker, scientist, or physician,” Gavin said. “They inspire us all to persevere in our shared mission to alleviate the physical, emotional, and financial strain that rare diseases place on too many individuals and families.”

The Community Champion, Policy Changemaker, Youth Leader, Lifetime Achievement and Abbey S. Meyers Leadership Awardees will be recognized in person at the NORD Breakthrough Summit® on Oct. 26-27 in Washington, D.C. The Scientific and Medical Trailblazer Awardees were recognized earlier this year at the NORD® Rare Disease Scientific Symposium, and NORD CEO Pamela Gavin is presenting the Industry Innovation Awards to each recipient company and their staff at their U.S. headquarters.

Learn more about the 2026 Rare Impact Awards at rareimpact.org.

About the National Organization for Rare Disorders (NORD^®)
Founded in 1983, the National Organization for Rare Disorders (NORD®) is the leading independent, nonpartisan, nonprofit organization dedicated to improving the health and lives of over 30 million Americans living with rare diseases. In partnership with more than 360 disease-specific NORD Member patient organizations and 49 NORD® Rare Disease Centers of Excellence spanning more than 170 medical institutions, NORD drives progress in rare disease research, care, and policy. Learn more at rarediseases.org.

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SOURCE National Organization for Rare Disorders (NORD®)