Despite remarkable advances in the understanding of the genetic foundations of inherited human diseases, treatment options continue to lag behind expectations. Nowhere is this more true than in the field of neuromuscular disease, including muscular dystrophy and other inherited muscle diseases. The only pharmacologic treatment that improves the natural history of such a disease is corticosteroids for Duchenne muscular dystrophy, and this treatment is by no means a cure or a definitive therapy. Thus, there is a great need for a pipeline of potential new therapies for muscular dystrophies and other inherited muscle diseases. Medosome Biotec, and its research partners at the University of Florida and Tufts Medical Center have created a new approach for screening drugs that could change the paradigm for the identification of promising new therapies for inherited muscle disease, and could help overcome some of the problems that have arisen with conventional screening methods. Several candidate drugs have been identified and are undergoing further testing.