Dr. Patricia Musolino of Mass General Brigham is pioneering gene editing approaches to treat Multisystem Smooth Muscle Dysfunction Syndrome (MSMDS)—a rare but devastating condition that causes stroke, heart attacks, and severe disability in children. In this episode, we explore how cutting-edge science is rewriting the prognosis for some of the sickest kids, and what it takes to translate genetic insight into real-world therapies under intense pressure.