Four years ago, Elizabeth Currid-Halkett received some of the worst news imaginable: her son, Eliot, was diagnosed with a fatal neuromuscular disease. In her quest to save her son, she learned of a gene therapy that ultimately helped Eliot, but she ran into roadblocks that made it difficult to access this life changing treatment. Her story, recently published in The New York Times, illustrates the obstacles that prevent some patients from obtaining rare disease treatments. In this episode, we are joined by Currid-Halkett, a USC Price School professor of public policy, and Alice Chen, an associate professor of public policy who recently testified before Congress about making rare disease treatments accessible.