New Directions in Biology and Disease of Skeletal Muscle

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DESCRIPTION (provided by applicant): The "New Directions in Biology and Disease of Skeletal Muscle" is an international symposium to be held June 17-20, 2012 at the Westin Canal Place in New Orleans, LA. The Muscular Dystrophy Care Act was passed into law in 2001 and reauthorized in 2008. One goal of the MD Care Act was to promote interaction between basic and clinical investigation to drive therapeutic development for the muscular dystrophies. In response, we organized the "New Directions" symposium. This is the fifth meeting of this series with prior meetings held in 2004, 2006, 2008 and 2010. The last meeting had 380 participants including 142 trainees. The 2010 meeting was held as a combined meeting with the Ottawa Neuromuscular Conference, and the post meeting survey confirmed the successful nature of this meeting. The muscular dystrophies are genetically diverse and share some pathological overlap with other degenerative and atrophic neuromuscular disorders. Although there is promise from proof-of-principle human clinical trials, there is presently no cure for these disorders, and treatment is largely supportive. Caring for patients with muscular dystrophy is at significant economic and emotional cost. Therefore, additional therapies must be conceived and implemented. There are at least four therapeutic goals for muscular dystrophy, and these include 1) Reduce muscle degeneration 2) Increase muscle regeneration 3) Improve respiratory muscle function 4) Prevent or treat cardiac muscle dysfunction when present. The molecular pathways to achieve these goals will be discussed with the goal of developing novel therapeutic strategies and/or planning for the transition of these ideas into preclinical or early phase human clinical trials. The scientific sessions will be devoted to inflammation and fibrosis in muscle disease, muscle growth and regeneration, disorders of trafficking in muscle, nuclear defects in muscle disease, signaling pathways for therapy, and gene correction/replacement strategies. The New Directions meeting will convene over 3.5 days with approximately 43 oral presentations and more than 100 poster presentations expected. Approximately 40% of the speakers will be determined by selection from submitted abstracts as to allow the inclusion of the most timely and current findings relevant to the field of the muscle degenerative diseases and to be inclusive of young investigators and trainees.