New Directions in Biology and Disease of Skeletal Muscle

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PROJECT SUMMARY This is a new proposal requesting partial support for "The New Directions in Biology and Disease of Skeletal Muscle," to be held July 6-9, 2020. The meeting will convene at the New York Marriott Marquis in Times Square. The “New Directions” initiated in 2004, and this meeting has taken place every other year, making the 2020 meeting the 9th in the series. The goal of this meeting is to promote translational science for neuromuscular disease, especially the muscular dystrophies. In 2004, at the time of the first meeting, there were no FDA approved drugs for neuromuscular disease and a limited pipeline with limited interest from the pharmaceutical industry and biotech. With the purpose of this meeting to bring together stakeholders including academic laboratories, small and large pharmaceutical interests, and patient/family advocacy groups, this meeting has been instrumental in promoting the identification and translation of new targets for neuromuscular disease. 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 target identification and translation of basic discovery into clinical trials for those with neuromuscular disease. The New Directions meeting differs from other muscle biology-related meetings because of inclusion of industry and academic attendees as well as heavy emphasis on trainee participation. Exon skipping drugs were approved for Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) in 2016 and 2017. Gene therapy for SMA in 2019 with approved as onasmemnogene abeparvovec, and there are multiple ongoing micro-dystrophin gene therapy trials for DMD. Based on this progress and on feedback from participants at the 2018 meeting, this next meeting will expand to other forms of inherited muscular diseases and focus on novel mechanisms for these disorders.