In recent years, a confluence of global economic, market, and policy shifts have led to the abandonment of more than 1,000 promising treatments in development for rare diseases - many of which deliver meaningful results for patients. Patient communities, researchers, and clinicians have had to pivot and figure out bold new ways of advancing or repurposing innovative treatments that traditional biopharma companies can no longer support. Session attendees will hear from the changemakers - patients, parents, advocates, researchers, clinicians, and industry leaders who refused to accept there wasn't a better way. The paths they are forging are not only changing the landscape for rare disease but paving the way for precision medicines for larger disease areas that are increasingly being segmented into collections of distinct, less common, and rare conditions.