Component C: Iowa MD STARnet Site Data Dissemination

组件 C：爱荷华州 MD STARnet 站点数据传播

• 批准号: 10002053
• 负责人: Paul A Romitti
• 金额: $ 8万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10002053
• 关键词: Component; Iowa; MD; STARnet; Site

PROJECT SUMMARY The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 authorized the Centers for Disease Control and Prevention to create a National Muscular Dystrophy Epidemiology Program. This program – The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) – was established in 2002 to conduct population-based surveillance and long-term follow-up of individuals diagnosed with Duchenne and Becker muscular dystrophies (DBMD). In 2011, the MD STARnet expanded to include cross-sectional surveillance for seven additional MDs (congenital, distal, Emery- Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal), and in 2014, this expanded surveillance was continued. Our Iowa MD STARnet Core Site was one of four charter awardees for the MD STARnet in 2002 and was successfully refunded in 2006, 2011, and 2014. As an MD STARnet Core Site, we successfully conducted population-based surveillance for DBMD and the additional MDs by integrating clinical, programmatic, and referral resources provided by key partners in Iowa. Our partnership with the Reproductive Molecular Epidemiology Research and Education Program at The University of Iowa has provided experienced investigators to analyze MD STARnet data and publish peer-reviewed manuscripts on priority MD STARnet data. For the proposed project, we will continue to use these experienced investigators to extend and expand our leadership role in analyzing and disseminating population-based surveillance and survey data for the nine MDs. We propose to continue working collaboratively with CDC investigators and other awarded grantees to disseminate research that focuses on high priority topic areas and informs on gaps in knowledge. To accomplish this, we aim to: 1) analyze surveillance data and disseminate research from high priority areas through peer-reviewed publications and presentations to target audiences; and 2) collaborate with CDC and awarded grantees on research projects. Among our planned research activities will be examining pregnancy among individuals with MD, evaluating clinical trial participation and non- participation, assessing the impact of clinical trial participation on the natural history of MD, and describing cardiopulmonary outcomes and treatments. Findings generated from these activities will inform on the public health impact of MD and contribute to the clinical understanding of the natural histories, survival and mortality, and healthcare needs of individuals and families affected by MD. These findings also will guide development and provision of policies and care programs to ensure access to comprehensive healthcare services and link individuals with MD and their families to social services that will improve quality of life.

项目摘要 2001年肌营养不良症社区援助，研究和教育修正案授权 疾病控制和预防中心创建了一个全国性的肌肉营养不良流行病学 程序.该计划-肌肉营养不良监测，跟踪和研究网络（MD STARnet）-于2002年成立，以人口为基础进行监测和长期随访， 被诊断患有杜氏和贝克肌营养不良症（DBMD）的个体。2011年，MD STARnet 扩展到包括对另外7个MD（先天性、远端、Emery- Dreifuss，面肩胛肱骨，肢带，肌强直和眼咽），并在2014年， 继续进行监视。我们的爱荷华州MD STARnet核心站点是MD的四个特许获奖者之一 STARnet于2002年推出，并于2006年，2011年和2014年成功退款。作为MD STARnet核心站点，我们 成功地进行了基于人群的DBMD和其他MD监测， 由爱荷华州的主要合作伙伴提供的临床、项目和转诊资源。我们与 爱荷华州大学的生殖分子流行病学研究和教育项目 提供经验丰富的研究人员分析MD STARnet数据，并在 优先MD STARnet数据。对于拟议的项目，我们将继续使用这些经验丰富的调查员 扩大和扩大我们在分析和传播基于人口的监测方面的领导作用， 九个MD的调查数据。我们建议继续与CDC调查人员合作， 其他获奖的受赠者传播研究，重点是高优先主题领域，并通报 知识差距。为了实现这一目标，我们的目标是：1）分析监测数据和传播研究 从高度优先领域，通过同行评审的出版物和向目标受众的介绍; 2） 与疾病预防控制中心合作，并在研究项目上获得资助。我们计划的研究活动将 检查MD患者的妊娠情况，评估临床试验参与情况， 参与，评估临床试验参与对MD自然史的影响，并描述 心肺结果和治疗。这些活动的结果将向公众提供信息 MD对健康的影响，并有助于临床理解的自然史，生存和 死亡率，以及受MD影响的个人和家庭的医疗保健需求。这些发现也将指导 制定和提供政策和护理方案，以确保获得全面的医疗保健 服务，并将MD患者及其家庭与社会服务联系起来，以提高生活质量。