Rare Breakthroughs: Hope Now and on the Horizon 

Our final plenary session of the day, “Rare Breakthroughs: Hope Now and on the Horizon,” was one to remember! Dr. PJ Brooks, NCATS (National Center for Advancing Translational Sciences) at NIH (National Institutes of Health), Dr. Peter Marks, CBER (Center for Biologics Evaluation and Research) at FDA (Food & Drug Administration), and Dr. Jennifer Cohen, Duke Children’s Hospital & Health Center left the audience with an immense amount of hope for the future of rare diseases. New innovations are helping to advance the diagnosis, treatment, and care of rare disease patients every day! The session highlighted current government initiatives and innovative technologies that can benefit rare disease patients and their families, now and in the future. Do you want to learn what is on the horizon? Click here to explore this session.