New Directions in Biology and Disease of Skeletal Muscle

描述(申请人提供)：“骨骼肌生物学和疾病的新方向”是一个国际研讨会，将于2012年6月17-20日在洛杉矶新奥尔良的威斯汀运河广场举行。肌肉营养不良护理法案于2001年通过成为法律，并于2008年重新授权。MD Care Act的目标之一是促进基础研究和临床研究之间的互动，以推动肌营养不良症的治疗开发。为此，我们组织了“新方向”研讨会。这是这一系列会议的第五次会议，前几次会议分别于2004年、2006年、2008年和2010年举行。上一次会议有380人参加，其中包括142名学员。2010年会议是作为渥太华神经肌肉会议的一次合并会议举行的，会后调查证实了这次会议的成功。肌营养不良是遗传多样性的，与其他退行性和萎缩性神经肌肉疾病有一些病理重叠。尽管原则验证的人类临床试验带来了希望，但目前还没有治愈这些疾病的方法，治疗在很大程度上是支持性的。照顾肌营养不良患者需要付出巨大的经济和情感代价。因此，必须构思和实施额外的治疗方法。肌营养不良症至少有四个治疗目标，包括1)减少肌肉退化，2)增加肌肉再生，3)改善呼吸肌功能，4)预防或治疗心肌功能障碍。将讨论实现这些目标的分子途径，目标是开发新的治疗策略和/或计划将这些想法转变为临床前或早期人类临床试验。科学会议将专门讨论肌肉疾病中的炎症和纤维化、肌肉生长和再生、肌肉运输障碍、肌肉疾病中的核缺陷、治疗的信号通路以及基因校正/替换策略。新方向会议将举行3.5天，预计将有大约43个口头陈述和100多个海报陈述。大约40%的发言者将从提交的摘要中挑选出来，以便纳入与肌肉退行性疾病领域有关的最及时和最新的发现，并包括年轻的研究人员和受训人员。

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.