New Directions in Biology and Disease of Skeletal Muscle

描述（由申请人提供）：这是一份新的提案，要求部分支持题为“骨骼肌生物学和疾病的新方向”的国际研讨会，将于2014年6月29日至7月2日在芝加哥市中心的芝加哥万豪酒店举行。“新方向”会议始于2004年，自2004年以来每两年举行一次。为了方便使用，会议轮换了会议地点，以前的会议地点包括圣地亚哥（2004年）、达拉斯（2006年）、安大略省渥太华（2010年）和新奥尔良（2008年和2012年）。在本次会议召开的十年中，神经肌肉疾病领域取得了显著的成功，将基础科学发现转化为罕见疾病患者的临床试验，包括肌肉萎缩症和脊髓性肌萎缩症。“新方向”会议不同于其他主题相关会议，因为它侧重于将工业界和学术界与会者聚集在一起，重点评估基于实验室的观察结果，评估或测试临床前和临床环境中治疗的适用性。本次会议的召开是为了响应MD保健法案，并认识到设计和测试罕见神经肌肉疾病的治疗方法需要所有利益相关者之间的组织和协调努力。除了注重确定和测试治疗途径外，“新方向”会议还高度重视学员和年轻研究人员的参与，因为人们认识到，这些医疗问题的挑战将需要进行多样化和长期的努力，以实现对这些毁灭性疾病的治疗。

DESCRIPTION (provided by applicant): 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, 2014 in Chicago IL at the Chicago Marriott Downtown. 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). Over the decade that this meeting has been held, the neuromuscular disease field has seen remarkable success in moving basic science discovery into the reality of clinical trials in patients with rare disorders including the muscular dystrophies and spinal muscular atrophy. The "New Directions" meeting differs from other topically related meetings because of its focus on bringing together industry and academic attendees with focus on evaluating laboratory based observations and assessing or testing suitability for therapy in the preclinical and clinical settig. This meeting was developed in response to the MD Care Act and the recognition 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.