National Organization for Rare Disorders (NORD) Announces 2025 Industry Innovation and Rare Impact Award Honorees

Annual Awards Celebrate Outstanding Contributions to Rare Disease Treatments, Advocacy, and Research

QUINCY, Mass., July 16, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) is honoring a distinguished group of industry leaders and individuals for their contributions to help improve the lives of more than 30 million Americans with a rare disease through the annual Industry Innovation and Rare Impact Awards.

The 2025 honorees include innovative companies whose treatments have significantly improved rare disease patient outcomes, community leaders raising awareness of rare disease, trailblazers in rare disease research, and advocates spearheading legislative change.

"With approximately 95% of 10,000 known rare disorders lacking approved treatments, it is vital that we come together to drive scientific breakthroughs," said Pamela K. Gavin, NORD Chief Executive Officer. "NORD is proud to honor these seven companies for pioneering new therapies and bringing hope to the rare disease community."

• argenx for VYVGART Hytrulo®: The first neonatal Fc receptor (FcRn) blocker approved for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare, debilitating, often progressive, neuromuscular disorder.
• Ascendis Pharma for YORVIPATH®: the first treatment for hypoparathyroidism, a rare endocrine disease, in adults.
• BridgeBio for Attruby^®: the first approved product with a label specifying near-complete stabilization of transthyretin (TTR) for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a rare progressive heart disease.
• Ionis Pharmaceuticals for TRYNGOLZA™: the first approved treatment to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
• PTC Therapeutics for KEBILIDI™: the first-ever gene therapy approved in the United States that is directly administered to the brain for the treatment of children and adults with Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder.
• Orchard Therapeutics for LENMELDY™: the first therapy of any kind for children with metachromatic leukodystrophy (MLD), a rare, inherited genetic disorder for which physicians have historically only been able to focus on symptom management.
• X4 Pharmaceuticals for XOLREMDI®: the first treatment to receive approval for persons with WHIM syndrome, a rare immunodeficiency disorder.

NORD is also proud to recognize the individuals and organizations dedicated to improving life for the one in 10 Americans living with a rare disorder, including:

• Hermansky-Pudlak Syndrome (HPS) Network, Inc: Winner of the Abbey S. Meyers Leadership Award, the HPS Network exemplifies extraordinary leadership in advocacy both locally, through the New York Rare Disease Advisory Council Coalition, and nationally, through its grassroots efforts in Puerto Rico.
• The Honorable Kevin Blackwell of Mississippi: Recognized with the Policy Changemaker Award, Mississippi State Sen. Kevin Blackwell sponsored the Mississippi Rare Disease Advisory Council (RDAC) bill that was enacted in 2024 and serves as an active member of its governing board, ensuring that rare disease patients throughout the state have a public voice.
• The Honorable Tammy Baldwin of Wisconsin: A Policy Changemaker Award recipient, U.S. Sen. Tammy Baldwin of Wisconsin has established herself as a steadfast champion for the rare disease community through sponsorship of the RARE Act and her consistent legislative leadership and bipartisan collaboration on critical health care initiatives.
• Robert Graham of New York: Community Champion honoree Bob Graham brings personal experience and passion to his advocacy work for the greater rare disease community in New York, both as a hemophilia patient and as a caregiver to family members with bleeding disorders.
• Jaime McHugh of New Hampshire: Recognized as a Community Champion and inspired by two daughters with rare diseases, Jaime McHugh is a leader in rare disease advocacy, raising awareness and funding as a key member of NORD's Running for Rare team.
• Dr. Angela Scheuerle of Texas: Recognized as a Medical & Scientific Trailblazer, Dr. Scheuerle's extensive, pioneering work in public health and drug safety at the University of Texas-Southwestern, a NORD® Rare Disease Center of Excellence, is a model of integrated clinical excellence, translational research, and mentorship — continually elevating quality of life for individuals living with rare diseases.
• Mary Nadon Scott of Vermont: Another Community Champion honoree, Mary Nadon Scott is a founding member of the Vermont Rare Disease Advisory Council Coalition, where she has been instrumental in building a strong, inclusive community that advocates for all those affected by rare diseases.
• Dr. Jerry Vockley of Pennsylvania: Also recognized as a Medical & Scientific Trailblazer, Dr. Vockley is the Co-Director of the NORD Rare Disease Center of Excellence at the University of Pittsburgh and UPMC Children's Hospital of Pittsburgh, where he has built a legacy of profound compassion, scientific innovation, and unwavering dedication to the rare disease community.

"The 2025 Rare Impact Award winners demonstrate the power we each have to drive change in our community, whether that's 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."

NORD will celebrate the extraordinary achievements in rare disease research, innovation, and advocacy by recognizing our 2025 honorees throughout the year in various ways, including expanded reception-style celebrations at our largest events. In this new format, honorees will benefit from broader visibility and recognition among peers and colleagues in the rare disease community.

• Community Champion, Policy Changemaker, Youth Leader, and Abby S. Meyers Leadership Awards will be celebrated during our annual NORD® Rare Diseases and Orphan Products Breakthrough Summit on Oct. 19-21 in Washington, D.C.
• Industry Innovation Awards will be presented to each recipient at their U.S. headquarters, where recognition will be shared with employees across various functions at the organization.
• Scientific and Medical Trailblazer Awardees were recognized at the NORD® Rare Disease Scientific Symposium on June 2-3 in Washington, D.C.

To learn more about the Rare Impact Award honorees, please visit rareimpact.org.

ABOUT THE NATIONAL ORGANIZATION FOR RARE DISORDERS
Founded in 1983, the National Organization for Rare Disorders (NORD®) is a 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 350 disease-specific member patient organizations, NORD drives progress in rare disease research, care, and policy. Learn more at rarediseases.org.

SOURCE National Organization for Rare Disorders (NORD®)