New Directions in Biology and Disease of Skeletal Muscle

This is a new proposal requesting partial support for an international symposium entitled "The New Directions in Biology and Disease of Skeletal Muscle," to be held June 29-July 2, 2016 in Orlando FL at the Renaissance Orlando at SeaWorld. The “New Directions” meeting began in 2004, and since 2004 this meeting has been held every other year. The meeting has rotated venues to permit accessibility, and prior meeting sites included San Diego (2004), Dallas (2006), Ottawa, Ontario (2010) and New Orleans (2008 and 2012), Chicago (2014) and now Orlando. This meeting was developed in response to the MD Care Act, which recognized a need for a neuromuscular disease focused meeting with specific emphasis on translation of basic discovery into clinical trials for those with neuromuscular disease. In the first New Directions meeting in 2004, we learned about exon skipping for Duchenne Muscular Dystrophy and toxic RNA as a mechanism in Myotonic Dystrophy. Because of interactions at this meeting, the field has progressed to clinical trials for these approaches. At the same time, there has been growing appreciation for the need to develop biomarkers of disease and to develop additional targeted approaches to treat neuromuscular disease. The New Directions meeting differs from other topically related meetings because of its focus on bringing together industry and academic attendees with focus evaluating laboratory based observations and assessing or testing suitability for therapy in the preclinical and clinical setting. This recognizes that devising and testing therapy for rare neuromuscular disorders requires organization and coordinated efforts among all stakeholders. In addition to the focus on identifying and testing therapeutic pathways, the New Directions meeting places a high emphasis on inclusion of trainees and young investigators, as it is recognized that the challenges of these medical problems will require a diverse and prolonged effort to realize cures for these devastating disorders.