NORD Announces 2025 Industry Innovation and Rare Impact Award Honorees

The National Organization for Rare Disorders (NORD) announced its 2025 Industry Innovation and Rare Impact Award honorees, recognizing seven companies for their treatments and contributions to rare disease research and advocacy. Honored companies include argenx, Ascendis Pharma, BridgeBio, Ionis Pharmaceuticals, PTC Therapeutics, Orchard Therapeutics, and X4 Pharmaceuticals. NORD also recognized individuals and organizations for their work in improving the lives of Americans with rare diseases.

The National Organization for Rare Disorders (NORD) has announced its 2025 Industry Innovation and Rare Impact Award honorees, recognizing seven companies for their significant contributions to rare disease treatments and advocacy. The awards celebrate innovative companies and individuals who have made substantial improvements in the lives of Americans living with rare diseases.

Among the honored companies are:
- Argenx for VYVGART Hytrulo®, the first neonatal Fc receptor (FcRn) blocker approved for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
- Ascendis Pharma for YORVIPATH®, the first treatment for hypoparathyroidism 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).
- 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 for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.
- Orchard Therapeutics for LENMELDY™, the first therapy for children with metachromatic leukodystrophy (MLD).
- X4 Pharmaceuticals for XOLREMDI®, the first treatment to receive approval for persons with WHIM syndrome.

NORD also recognized several individuals and organizations for their exceptional work in improving the lives of Americans with rare diseases. These include:
- The Hermansky-Pudlak Syndrome (HPS) Network, Inc., which won the Abbey S. Meyers Leadership Award.
- The Honorable Kevin Blackwell of Mississippi, recognized with the Policy Changemaker Award.
- The Honorable Tammy Baldwin of Wisconsin, also a Policy Changemaker Award recipient.
- Robert Graham of New York, a Community Champion honoree.
- Jaime McHugh of New Hampshire, another Community Champion honoree.
- Dr. Angela Scheuerle of Texas, recognized as a Medical & Scientific Trailblazer.
- Mary Nadon Scott of Vermont, another Community Champion honoree.
- Dr. Jerry Vockley of Pennsylvania, also recognized as a Medical & Scientific Trailblazer.

The 2025 Rare Impact Award winners demonstrate the power of collective effort in driving change within the rare disease community. NORD will celebrate these achievements throughout the year, including during its annual NORD® Rare Diseases and Orphan Products Breakthrough Summit on Oct. 19-21 in Washington, D.C.

References:
[1] https://www.prnewswire.com/news-releases/national-organization-for-rare-disorders-nord-announces-2025-industry-innovation-and-rare-impact-award-honorees-302505892.html
[2] https://www.openpr.com/news/4103066/angptl3-therapy-market-rises-with-focus-on-cardiovascular