Our goal is to develop next generation treatments for diseases in children. Our particular focus is on genetic diseases of muscle (such as Duchenne muscular dystrophy), but by targeting the underlying molecular biology of disease we believe we can help a broad array of human illnesses. Through our understanding of drug pathways, we are also developing serum-based biomarkers reflective of drug activities in patients. Ultimately, we feel this approach has the power to greatly improve the treatment of a large number of diseases featuring chronic inflammation, muscle disease or heart failure.