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Therapeutic Targets for the Congenital Muscular Dystrophies

先天性肌营养不良症的治疗目标

基本信息

批准号：
8153397
负责人：
CARSTEN G BONNEMANN
金额：
$ 1.5万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2009
资助国家：
美国
起止时间：
2009-07-01 至 2010-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8153397
关键词：
Academia Address Advocacy Animal Model Antibodies Area Attention Awareness Basic Science Biological Assay Childhood Clinical Clinical Treatment Clinical Trials Clinical Trials Design Communities Consensus Development Education Educational workshop Fellowship Fibroblasts Focus Groups Fostering Funding Funding Agency Future Goals Grant Industry Investments Knowledge Lead Methods Morbidity - disease rate Muscle Outcome Outcome Measure Pharmaceutical Resources Play Qualifying Rare Diseases Readiness Registries Research Research Personnel Research Support Resources Role Specimen Translating Translational Research Translations United States National Institutes of Health Validation base congenital muscular dystrophy design interest neuromuscular patient population pre-clinical public health relevance success symposium therapeutic target tool treatment strategy treatment trial working group

项目摘要

DESCRIPTION (provided by applicant): This proposal seeks funding for a unique conference devoted to identifying and initiating opportunities for translational research in the congenital muscular dystrophies (CMDs). The CMDs as a group are a significant contributor to childhood onset neuromuscular morbidity, but have received relatively little attention in neuromuscular research. The primary goal of the conference is to address, evaluate and achieve consensus on therapeutic targets in the CMDs by bringing together experts ranging from basic science to clinical trial design in rare disorders, as well as representatives from industry, advocacy and funding agencies (NIH and MDA). A successful outcome will lead to the construction of a roadmap and investment "portfolio" based on the identification of short term, mid term and long range scientific targets and their subsequent development into treatment strategies with strong translational potential towards clinical trials. This conference will be co-sponsored by Cure CMD, a nonprofit advocacy group focused on awareness, education and research support for translational targets in the CMDs.

描述（由申请人提供）：该提案旨在为一个独特的会议提供资金，该会议致力于识别和启动先天性肌营养不良症（CMD）转化研究的机会。CMD作为一个群体是儿童期神经肌肉发病率的重要贡献者，但在神经肌肉研究中受到的关注相对较少。会议的主要目标是通过汇集从基础科学到罕见疾病临床试验设计的专家，以及来自行业，倡导和资助机构（NIH和MDA）的代表，解决，评估和达成关于CMD治疗目标的共识。一个成功的结果将导致建立一个路线图和投资“组合”的基础上确定短期，中期和长期的科学目标，并将其随后发展为治疗策略，具有强大的转化潜力，以临床试验。本次会议将由Cure CMD共同主办，Cure CMD是一个非营利性倡导组织，专注于对CMD中转化目标的认识、教育和研究支持。