Meeting of the Minds 2024

As we prepare to welcome families to the CureDuchenne FUTURES Annual Conference, scientific leaders, executives from nearly every pharmaceutical and biotech company focused on developing therapies for Duchenne and Becker muscular dystrophy, and regulatory leaders came together for a discussion on addressing the challenges and opportunities in bringing treatments to individuals who need them. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER), and Peter Stein, M.D., the Director of CDER’s Office of New Drugs (OND), at the U.S. Food & Drug Administration spoke to attendees on rare disease gene and cell therapy development in the era of approved therapies.