Neurology
Muscular Dystrophy Translational Research Program
Our translational research program focuses on accelerating genetic precision therapies for the muscular dystrophies. We focus on understanding the pathogenesis of congenital myotonic dystrophy in the lab as well as modifiers of disease severity in myotonic dystrophy. We are working to reduce genetic variants of unknown significance in limb girdle muscular dystrophy. In myotonic dystrophy and limb girdle muscular dystrophy we lead international networks dedicated to understanding the natural history of these conditions.
Neurosciences