NORD Honors 2026 Trailblazers in Rare Disease Innovation and Advocacy

This year’s industry accolades spotlight six organizations that secured critical FDA approvals for first-of-their-kind therapies. Among those recognized are Mighty Therapeutics for its work on Barth syndrome, UCB for advancements in thymidine kinase 2 deficiency treatment, and Fondazione Telethon, which achieved a milestone as the first nonprofit to develop an FDA-approved gene therapy for Wiskott-Aldrich syndrome. Other honorees include Omeros, Jazz Pharmaceuticals, and Verastem Oncology, each addressing significant gaps in treatment for conditions ranging from aggressive brain tumors to rare blood disorders.

Beyond corporate breakthroughs, NORD highlighted the human element of medical progress. Awards were presented to advocates such as Dr. Stephanie E. Haridopolos, who successfully lobbied for the inclusion of Duchenne muscular dystrophy in national newborn screening panels, and Mark Skinner, who received the Lifetime Achievement Award for his global impact on bleeding disorders advocacy. These individuals, alongside youth leaders and medical educators, represent a multi-layered approach to patient care. NORD CEO Pamela Gavin noted that while 95% of the 10,000 known rare diseases still lack an approved treatment, these honorees provide the essential momentum required to expand the boundaries of what is medically possible.