The muscular dystrophy coordinating committee action plan for the muscular dystrophies.

The muscular dystrophy research community is at a watershed moment. Our understanding of the molecular, cellular, and physiological underpin­ nings of the muscular dystrophies continues to increase and has revealed several therapeutic tar­ gets. For the first time, investigational therapies are reaching the final stages of review by United States regulatory agencies. Yet, as we learn more about these diseases and the ongoing efforts to treat them, we are more aware of the critical work that remains. In this editorial we describe the work of the collaborative Muscular Dystrophy Coordinat­ ing Committee (MDCC), a congressionally man­ dated consortium of federal agencies and patient advocates tasked with identifying the many chal­ lenges confronting patients and their families and coordinating efforts to address them. Recently, the MDCC released an updated version of its Action Plan for the Muscular Dystrophies, a comprehensive document outlining the needs and priorities of the muscular dystrophy patient, family, and research communities. Our hope for the scientific community is that it will facilitate strategic planning and identifi­ cation of common goals among funders, encourage researchers both inside and outside of the muscular dystrophy field to investigate the Plan’s focus areas, and foster activities to further strengthen the train­ ing and career development of the next generation of researchers in the muscular dystrophies. The muscular dystrophies comprise a heteroge­ neous group of genetic disorders that involve pro­ gressive weakness and degeneration of skeletal muscle. There are dozens of genetically distinct types. Most also involve cardiac and smooth muscle, and some involve the brain, skeleton, and