NORD Honors Six Firms for First-of-Kind Rare Disease Therapy Approvals
Norwell, Mass. – September 24, 2026 -- The National Organization for Rare Disorders (NORD) has named six companies and nonprofits as 2026 Industry Innovator honorees for delivering first-of-their-kind FDA-approved therapies to patients with rare diseases that affect a fraction of the more than 30 million Americans living with such conditions.
Six FDA approvals mark milestones across ultra-rare indications
Mighty Therapeutics was recognized for FORZINITY, the first therapy for Barth syndrome, a rare mitochondrial disease, shown to improve muscle strength and quality of life. UCB earned recognition for KYGEVVI, the first and only approved treatment for thymidine kinase 2 deficiency in patients whose symptoms began at or before age 12. Fondazione Telethon was honored for Waskyra, a gene therapy for Wiskott-Aldrich syndrome that stands as the first FDA-approved gene therapy for a primary immunodeficiency disorder and the first developed by a nonprofit.