Therapeutic Targets for the Congenital Muscular Dystrophies

描述（由申请人提供）：本提案为一个独特的会议寻求资金，该会议致力于确定和启动先天性肌营养不良症（cmd）的转化研究机会。CMDs作为一个群体是儿童发病神经肌肉疾病的重要贡献者，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，讨论、评估和达成关于CMDs治疗目标的共识。一个成功的结果将导致在确定短期、中期和长期科学目标的基础上构建路线图和投资“组合”，并随后将其发展成具有强大临床试验转化潜力的治疗策略。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转化目标的认识、教育和研究支持。

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.