NORD Recognizes Leaders Advancing Rare Disease Innovation and Care
The National Organization for Rare Disorders (NORD) has announced the recipients of its 2026 Rare Impact Awards, honoring those who have made significant contributions to innovation, research, and advocacy for rare diseases.
Norwell, Massachusetts – The National Organization for Rare Disorders (NORD) has unveiled the honorees for its 2026 Rare Impact Awards. The awards recognize individuals and organizations for their substantial advancements in improving the lives of the more than 30 million Americans living with rare diseases.
This year, six companies and one nonprofit organization are being celebrated for developing novel therapies that have significantly improved patient outcomes. These honorees have also pioneered new research approaches and advanced advocacy efforts, contributing to a broader understanding and treatment landscape for rare conditions.
Among the recognized companies are Mighty Therapeutics for FORZINITY™, the first therapy for Barth syndrome; UCB for KYGEVVI, a treatment for thymidine kinase 2 deficiency; and Fondazione Telethon for Waskyra™, the first FDA-approved gene therapy for a primary immunodeficiency disorder. Other honorees include Omeros for YARTEMLEA®, Jazz Pharmaceuticals for MODEYSO™, and Verastem Oncology for AVMAPKI FARCYNJA CO-PACK.
Additionally, the Foundation for Prader-Willi Research (FPWR) received the Abbey S. Meyers Leadership Award for its patient-centered research initiatives and the development of the Global PWS Registry. This registry played a role in the FDA approval of a rare disease therapy developed via NORD's IAMRARE platform.