In celebration of 2026 Rare Disease Day, nearly 2,300 researchers, clinicians, patients, and advocates filled the halls of the NIH Natcher Conference Center or joined virtually to highlight the urgency and opportunity of advancing rare disease research. With more than 10,000 identified rare diseases affecting millions of Americans, speakers emphasized the need for innovative trial design, cross-sector collaboration, and scalable genomic approaches to accelerate diagnosis and treatment.
