Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific M

先天性肌营养不良症：从临床病理学到基础科学 M

• 批准号: 8319246
• 负责人: DEAN J. BURKIN
• 金额: $ 2.85万
• 依托单位: UNIVERSITY OF NEVADA RENO
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-04-01 至 2013-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8319246
• 关键词: Address; Advocacy; Animal Model; Apoptosis; Area; Basic Science; Biochemical; Biological Assay; Biomechanics; Cell Communication; Cell Differentiation process; Clinical; Clinical Pathology; Collaborations; Collagen Type VI; Connective Tissue; Consensus; Contracture; Cytoskeleton; Development; Disease; Disease model; Drug Industry; Educational workshop; Extracellular Matrix; Extracellular Matrix Proteins; Fibrosis; Funding; Future; Generations; Genetic; Genotype; Goals; Inflammation; International; Knowledge; Laminin; Lead; Life; Longevity; Mediator of activation protein; Membrane; Modeling; Modification; Molecular; Muscle; Muscular Dystrophies; Mutation; Myocardium; Myopathy; Natural regeneration; Neuraxis; Neuromuscular Junction; Nevada; Organelles; Outcome; Pathogenesis; Pathology; Pathway interactions; Patients; Pharmacologic Substance; Phenotype; Play; Process; Quality of life; Receptor Signaling; Relative (related person); Research; Research Personnel; Role; Scientist; Screening procedure; Signal Transduction; Skeletal Muscle; Therapeutics for Rare and Neglected Diseases; Translational Research; United States National Institutes of Health; Universities; alpha Dystroglycan; biobank; comparative; congenital muscular dystrophy; design; drug discovery; high throughput screening; interest; lens; link protein; medical schools; mitochondrial dysfunction; muscle regeneration; neuromuscular; novel; patient registry; physical property; research and development; satellite cell; stem cell niche; symposium; therapeutic development; therapeutic target; therapy development

DESCRIPTION (provided by applicant): This application seeks NIH funding to support a conference titled "Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific Mechanisms, Exploring the Role of the Myomatrix" which will be held at the University of Nevada School of Medicine from Sunday April 22 to Tuesday April 24, 2012. This conference will bring together basic scientists, clinicians, advocacy groups and pharmaceutical industry representatives with expertise in the matrix and muscular dystrophies in general, those with expertise in forms of CMD more specifically, and those from outside the immediate neuromuscular field but with expertise that is relevant to the CMDs. This is a unique conference dedicated to the intersection of muscle and extracellular matrix both in development and disease states, specifically muscular dystrophy and CMD. The conference will combine two keynote addresses with seven core sessions: matrix in development, matrix and regeneration, matrix and intracellular organelles, matrix biomechanical and physical properties, matrix and signaling receptors, matrix and the neuromuscular junction. The conference will end with a panel discussion to build a myomatrix roadmap, unifying key themes from prior sessions, providing opportunities for productive collaborations and identifying pathways towards translational research for the congenital muscular dystrophies. PUBLIC HEALTH RELEVANCE: This application seeks funding to support a conference titled "Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific Mechanisms, Exploring the Role of the Myomatrix". The primary conference goal is to address, evaluate and achieve consensus on contributing mechanisms of the myomatrix that drive key clinical pathology relevant to the quality of life and life span of patients with congenital muscular dystrophies. Conference deliverables include defining new focus areas, bringing together researchers that may otherwise not have a chance to communicate or collaborate and using the congenital muscular dystrophy clinical context to frame research queries into translational targets.

描述（由申请人提供）：本申请寻求NIH资助，以支持将于2012年4月22日（星期日）至4月24日（星期二）在内华达州医学院举行的题为“先天性肌营养不良：从临床病理学到潜在的科学机制，探索肌基质的作用”的会议。本次会议将汇集基础科学家，临床医生，倡导团体和制药行业的代表与矩阵和肌营养不良症的一般专业知识，那些在CMD形式更具体的专业知识，以及那些来自直接神经肌肉领域以外，但与CMD相关的专业知识。这是一个独特的会议，致力于肌肉和细胞外基质在发展和疾病状态，特别是肌肉萎缩症和CMD的交叉。会议将结合联合收割机两个主题演讲与七个核心会议：矩阵的发展，矩阵和再生，矩阵和细胞内细胞器，矩阵的生物力学和物理特性，矩阵和信号受体，矩阵和神经肌肉接头。会议将以小组讨论结束，以建立一个肌基质路线图，统一以前会议的关键主题，为富有成效的合作提供机会，并确定先天性肌营养不良症转化研究的途径。 公共卫生相关性：该申请寻求资金支持一个题为“先天性肌营养不良症：从临床病理学到潜在的科学机制，探索肌基质的作用”的会议。会议的主要目标是解决，评估和达成共识，促进机制的肌基质，推动关键临床病理学相关的生活质量和寿命的先天性肌营养不良症患者。会议成果包括定义新的重点领域，汇集研究人员，否则可能没有机会沟通或合作，并使用先天性肌营养不良症的临床背景框架研究查询转化为目标。