I enjoyed delivering the keynote address at the ninth-annual Hope on the Hill Congressional Dinner, hosted by Cure Spinal Muscular Atrophy (SMA). This group of hereditary neurodegenerative disorders destroys neurons in the spinal cord, leading to progressively debilitating muscle wasting. The dinner brings together SMA families, government officials, and industry leaders to celebrate progress towards curing these conditions. While at the dinner, I got to say hello to young Evelyn Villareal. Evelyn, who was born with SMA, is a gene therapy success story and an inspiration to us all. Hope on the Hill took place on December 3, 2019 at the Willard Intercontinental Hotel, Washington, D.C. Credit: NIH
