New Directions in Biology and Disease of Skeletal Muscle

项目摘要 这是一个新的建议，要求部分支持“新方向的生物学和疾病的skeleton 肌肉，“将于2020年7月6日至9日举行。会议将在时代广场的纽约马奎斯万豪酒店召开 广场2004年发起的“新方向”，这一会议隔年举行一次，使得 2020年是该系列的第九次会议。本次会议的目标是促进翻译科学， 神经肌肉疾病，尤其是肌肉萎缩症。2004年，在第一次会议上， 没有FDA批准的用于神经肌肉疾病的药物， 制药业和生物技术。本次会议的目的是召集利益攸关方， 学术实验室，小型和大型制药利益，以及患者/家庭倡导团体，这 会议在促进神经肌肉疾病新靶点的识别和转化方面发挥了重要作用。 疾病这次会议是为了响应《医学博士护理法案》而召开的，该法案认识到需要 神经肌肉疾病重点会议，特别强调目标识别和基本翻译 用于神经肌肉疾病患者的临床试验。新方向会议不同于 其他肌肉生物学相关的会议，因为包括工业和学术与会者，以及 非常重视学员的参与。外显子跳跃药物被批准用于Duchenne肌肉 营养不良（DMD）和脊髓性肌萎缩症（SMA）在2016年和2017年。2019年SMA的基因治疗， 批准为onasmemnogene abeparvovec，并且有多种正在进行的微肌营养不良蛋白基因治疗 DMD的临床试验根据这一进展以及2018年会议与会者的反馈， 会议将扩大到其他形式的遗传性肌肉疾病，并集中在新的机制，这些 紊乱

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.