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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：
8400254
负责人：
Elizabeth M McNally
金额：
$ 3.25万
依托单位：
UNIVERSITY OF CHICAGO
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-06-20 至 2013-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8400254
关键词：
Academia Access to Information Advocacy Atrophic Biology Caring Clinical Clinical Investigator Clinical Trials Collaborations Data Defect Degenerative Disorder Disease Doctor of Philosophy Economics Emotional Ensure Fibrosis Functional disorder Funding Genes Goals Government Grant Growth Hereditary Disease Human Industry Inflammation International Laws Membrane Mitochondrial Diseases Molecular Muscle Muscle Weakness Muscle function Muscular Atrophy Muscular Dystrophies Myocardium Myopathy Natural regeneration Nature Neuromuscular Diseases Nuclear Nuclear Envelope Ontario Oral Participant Pathogenesis Pathway interactions Patient Care Phase Private Sector Publishing Request for Proposals Research Personnel Respiratory Muscles Schedule Scientist Series Signal Pathway Signal Transduction Site Skeletal Muscle Students Surveys Therapeutic Time Translations Travel Work abstracting cost design gene correction gene replacement improved instructor interest meetings muscle degeneration muscle regeneration neuromuscular novel therapeutics patient advocacy group posters pre-clinical prevent professor programs repaired response sarcopenia symposium therapeutic development therapy development trafficking transmission process unpublished works

项目摘要

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.

描述（由申请人提供）：“骨骼肌生物学和疾病的新方向”是一次国际研讨会，将于 2012 年 6 月 17 日至 20 日在路易斯安那州新奥尔良威斯汀运河广场举行。《肌营养不良症护理法案》于 2001 年通过成为法律，并于 2008 年重新授权。MD 护理法案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治疗的发展。为此，我们组织了“新方向”研讨会。这是该系列会议的第五次会议，此前曾于2004年、2006年、2008年和2010年举行过会议。上一次会议有380人参加，其中142名学员。 2010年会议是与渥太华神经肌肉会议合并举行的，会后调查证实了这次会议的成功性质。肌营养不良症具有遗传多样性，并且与其他退行性和萎缩性神经肌肉疾病有一些病理重叠。尽管原理验证人体临床试验有希望，但目前还无法治愈这些疾病，并且治疗在很大程度上是支持性的。照顾肌营养不良症患者需要付出巨大的经济和情感成本。因此，必须构思和实施额外的疗法。肌营养不良症至少有四个治疗目标，其中包括 1) 减少肌肉退化 2) 增加肌肉再生 3) 改善呼吸肌功能 4) 预防或治疗存在的心肌功能障碍。将讨论实现这些目标的分子途径，目的是开发新的治疗策略和/或规划将这些想法转变为临床前或早期人体临床试验。科学会议将致力于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、治疗信号通路以及基因校正/替换策略。新方向会议将召开超过 3.5 天，预计将进行约 43 场口头演讲和 100 多场海报演讲。大约 40% 的演讲者将从提交的摘要中选择确定，以便纳入与肌肉退行性疾病领域相关的最及时、最新的发现，并包括年轻的研究人员和学员。