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New Directions in Biology and Disease of Skeletal Muscle

骨骼肌生物学和疾病的新方向

基本信息

批准号：
8720398
负责人：
Elizabeth M McNally
金额：
$ 3万
依托单位：
UNIVERSITY OF CHICAGO
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-04-01 至 2015-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8720398
关键词：
Academia Access to Information Advocacy Am 80 Basic Science Biology Caring Charge Chicago Clinical Clinical Investigator Clinical Trials Collaborations Consensus Data Degenerative Disorder Development Disease Doctor of Philosophy Elements Funding Government Grant Industry Inherited International Laboratories Medical Muscle Muscular Dystrophies Mutation Myopathy Myotonic Dystrophy Nature Neuromuscular Diseases Nuclear Envelope Ontario Oral Outcome Participant Pathway interactions Patients Preclinical Testing Private Sector Publications Rare Diseases Request for Proposals Research Research Personnel Site Skeletal Muscle Spinal Muscular Atrophy Students Testing Therapeutic Time Translations Travel abstracting base design exon skipping expectation improved instructor meetings patient advocacy group posters pre-clinical professor programs public health relevance research clinical testing response success symposium therapy development trend unpublished works

项目摘要

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.

描述(由申请人提供)：这是一项新的提案，请求部分支持将于2014年6月29日至7月2日在芝加哥万豪市中心举行的名为“骨骼肌生物学和疾病的新方向”的国际研讨会。“新方向”会议始于2004年，自2004年以来每隔一年举行一次。会议轮换了地点，以便于进入，之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)。在举行这次会议的十年里，神经肌肉疾病领域在将基础科学发现转化为临床试验方面取得了显著成功，这些患者患有包括肌肉营养不良和脊髓性肌萎缩在内的罕见疾病。“新方向”会议不同于其他与主题相关的会议，因为它的重点是将产业界和学术界的与会者聚集在一起，重点评估基于实验室的观察结果，并评估或测试临床前和临床环境中治疗的适宜性。这次会议是为了响应MD Care Act，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的组织和协调努力而召开的。除了注重确定和测试治疗途径外，“新方向”会议高度强调纳入受训人员和青年研究人员，因为人们认识到，这些医学问题的挑战将需要作出各种和长期的努力，以实现对这些破坏性疾病的治疗。