Anna O. Jesus, MD and Rebecca J. Scharf, MD, both neurodevelopmental pediatricians at UVA Children’s, discuss how gene therapy is used to treat Spinal Muscular Atrophy (SMA) in the video published recently by UVA Today. The story follows one family in their journey to treat the potentially fatal disease in their daughter Isabelle. She has responded well to gene therapy treatment and is thriving.
Watch the video “The Life-Saving Power of Gene Therapy.”