NORD Honors Pioneers of Rare Disease Research and Advocacy for 2025 Awards
The National Organization for Rare Disorders (NORD) recently unveiled its 2025 Industry Innovation and Rare Impact Award winners, celebrating exceptional leaders and organizations that have made significant contributions towards improving the lives of the over 30 million Americans battling rare diseases. These awards aim to honor those who have pioneered treatments, advocated for legislative change, or raised awareness regarding the often-overlooked realm of rare disorders.
Among the recipients, companies like argenx, Ascendis Pharma, and BridgeBio stood out for their innovative approaches in treating rare diseases. argenx's VYVGART Hytrulo® has become a vital treatment for patients suffering from chronic inflammatory demyelinating polyneuropathy (CIDP), illustrating the need for breakthroughs in rare neuromuscular disorders. Ascendis Pharma received accolades for YORVIPATH®, the first targeted therapy for hypoparathyroidism, thereby showcasing its commitment to addressing rare endocrine conditions.
Similarly, BridgeBio's product Attruby® has made waves by stabilizing transthyretin (TTR) in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), marking a crucial advancement for patients battling this rare heart condition. Ionis Pharmaceuticals also made the list with TRYNGOLZA™, recognized as the first approved treatment for reducing triglyceride levels in individuals with familial chylomicronemia syndrome (FCS), demonstrating the critical need for management options in this rare genetic disorder.
The 2025 awards also highlighted the first-ever gene therapy from PTC Therapeutics for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, underscoring the rapid advancements being made in the gene therapy landscape. Furthermore, Orchard Therapeutics celebrated a significant milestone with LENMELDY™, aimed at treating children with metachromatic leukodystrophy (MLD), a genetic disorder that has historically seen limited treatment options. X4 Pharmaceuticals rounded out the prestigious group with XOLREMDI®, the first treatment approved for WHIM syndrome, a rare immune disorder, demonstrating pioneering efforts in immunology.
In addition to organizational achievements, NORD recognized impactful individuals in the rare disease community. The Hermansky-Pudlak Syndrome (HPS) Network, Inc. was honored with the Abbey S. Meyers Leadership Award for their exceptional advocacy efforts, raising awareness locally and nationally. Policy Changemaker Awards were presented to significant legislative figures such as Mississippi State Senator Kevin Blackwell and U.S. Senator Tammy Baldwin from Wisconsin, both of whom have shown unwavering dedication to supporting individuals with rare diseases through legislative measures.
Community champions like Robert Graham and Jaime McHugh were celebrated for their commitment to advocating for rare disease awareness and funding. Dr. Angela Scheuerle and Dr. Jerry Vockley, both trailblazers in medical research, were also recognized for their groundbreaking work in improving patient quality of life by pushing the boundaries of science and healthcare.
Pamela K. Gavin, CEO of NORD, summarized the importance of these recognitions, emphasizing that the winners' endeavors inspire collective action towards alleviating the challenges faced by those with rare diseases. According to Gavin, "The accolades our honorees receive represent a collective hope as we strive to enhance the healthcare landscape for those impacted by rare conditions."
Throughout 2025, NORD will celebrate these achievements across various platforms and events, ensuring that honorees receive the recognition they deserve within the rare disease community. Significant celebrations will culminate in events like the NORD® Rare Diseases and Orphan Products Breakthrough Summit scheduled for October 19-21 in Washington, D.C., where community champions and policy leaders will come together to foster collaboration.
NORD, established in 1983, has been dedicated to improving the health and lives of Americans living with rare diseases and will continue to support and promote the essential work of these award-winning organizations and individuals. To learn more about their efforts and the 2025 award honorees, visit rareimpact.org.
Among the recipients, companies like argenx, Ascendis Pharma, and BridgeBio stood out for their innovative approaches in treating rare diseases. argenx's VYVGART Hytrulo® has become a vital treatment for patients suffering from chronic inflammatory demyelinating polyneuropathy (CIDP), illustrating the need for breakthroughs in rare neuromuscular disorders. Ascendis Pharma received accolades for YORVIPATH®, the first targeted therapy for hypoparathyroidism, thereby showcasing its commitment to addressing rare endocrine conditions.
Similarly, BridgeBio's product Attruby® has made waves by stabilizing transthyretin (TTR) in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), marking a crucial advancement for patients battling this rare heart condition. Ionis Pharmaceuticals also made the list with TRYNGOLZA™, recognized as the first approved treatment for reducing triglyceride levels in individuals with familial chylomicronemia syndrome (FCS), demonstrating the critical need for management options in this rare genetic disorder.
The 2025 awards also highlighted the first-ever gene therapy from PTC Therapeutics for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, underscoring the rapid advancements being made in the gene therapy landscape. Furthermore, Orchard Therapeutics celebrated a significant milestone with LENMELDY™, aimed at treating children with metachromatic leukodystrophy (MLD), a genetic disorder that has historically seen limited treatment options. X4 Pharmaceuticals rounded out the prestigious group with XOLREMDI®, the first treatment approved for WHIM syndrome, a rare immune disorder, demonstrating pioneering efforts in immunology.
In addition to organizational achievements, NORD recognized impactful individuals in the rare disease community. The Hermansky-Pudlak Syndrome (HPS) Network, Inc. was honored with the Abbey S. Meyers Leadership Award for their exceptional advocacy efforts, raising awareness locally and nationally. Policy Changemaker Awards were presented to significant legislative figures such as Mississippi State Senator Kevin Blackwell and U.S. Senator Tammy Baldwin from Wisconsin, both of whom have shown unwavering dedication to supporting individuals with rare diseases through legislative measures.
Community champions like Robert Graham and Jaime McHugh were celebrated for their commitment to advocating for rare disease awareness and funding. Dr. Angela Scheuerle and Dr. Jerry Vockley, both trailblazers in medical research, were also recognized for their groundbreaking work in improving patient quality of life by pushing the boundaries of science and healthcare.
Pamela K. Gavin, CEO of NORD, summarized the importance of these recognitions, emphasizing that the winners' endeavors inspire collective action towards alleviating the challenges faced by those with rare diseases. According to Gavin, "The accolades our honorees receive represent a collective hope as we strive to enhance the healthcare landscape for those impacted by rare conditions."
Throughout 2025, NORD will celebrate these achievements across various platforms and events, ensuring that honorees receive the recognition they deserve within the rare disease community. Significant celebrations will culminate in events like the NORD® Rare Diseases and Orphan Products Breakthrough Summit scheduled for October 19-21 in Washington, D.C., where community champions and policy leaders will come together to foster collaboration.
NORD, established in 1983, has been dedicated to improving the health and lives of Americans living with rare diseases and will continue to support and promote the essential work of these award-winning organizations and individuals. To learn more about their efforts and the 2025 award honorees, visit rareimpact.org.
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