NeURoscience is a quarterly newsletter of the Del Monte Institute for Neuroscience at the University of Rochester. It highlights neuroscience research, grants, faculty, and students. In this issue, read about how a half-century journey toward genetic therapies in muscular dystrophy at the Medical Center has played a pivotal role in efforts to understand the complex progression and underlying genetic mechanisms of myotonic dystrophy, Duchenne muscular dystrophy, and other rare neuromuscular disorders. This work has created an international network of researchers and resources necessary to bring new treatments to market. This long history and depth of expertise puts URMC researchers at the center of efforts to accelerate the study and approval of a growing wave of genetic therapies for neuromuscular disorders that are revolutionizing care.