Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

Overview

Duchenne muscular dystrophy (DMD) results from the absence of dystrophin, leading to membrane injury and a breakdown of muscle fibers. This study investigates the role of fast skeletal muscle contraction in DMD using an orally active inhibitor of fast skeletal muscle myosin, EDG-5506. Even modest reductions in contraction (<15%) are found to protect skeletal muscles in dystrophic mice from stress injury, with long-term treatment decreasing muscle fibrosis. Importantly, therapeutic levels of myosin inhibition with EDG-5506 do not negatively impact strength or coordination. This unexpected finding suggests a potential novel treatment strategy for DMD and related myopathies.