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: 10441052
• 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/10441052
• 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.

项目总结 2001年批准的肌营养不良症社区援助、研究和教育修正案 美国疾病控制与预防中心创建国家级肌营养不良流行病学 程序。该计划-肌肉营养不良监测、跟踪和研究网络(MD STARnet)-成立于2002年，目的是进行以人口为基础的监测和长期跟踪 被诊断为Duchenne和Becker肌营养不良症(DBMD)的个人。2011年，MD星网 扩大到包括对另外七个MD的横断面监测(先天性、远端、砂砾- Dreifuss、面部肩肱、四肢腰带、肌强直和眼咽部)，并在2014年扩大 监视仍在继续。我们的爱荷华州MD星网核心站点是MD的四个特许权获得者之一 并在2006、2011和2014年成功退款。作为MD STARnet核心站点，我们 成功地对DBMD和额外的MD进行了以人口为基础的监测 爱荷华州主要合作伙伴提供的临床、方案和转诊资源。我们的合作伙伴关系 爱荷华大学生殖分子流行病学研究和教育项目 提供经验丰富的调查人员分析MD STARnet数据并在以下网站上发表同行评审的手稿 优先MD星网数据。对于拟议的项目，我们将继续使用这些经验丰富的调查人员 扩大我们在分析和传播以人口为基础的监测和宣传方面的领导作用 九位MD的调查数据。我们建议继续与疾控中心调查人员合作，并 其他获奖者传播侧重于高优先主题领域的研究，并就 知识上的差距。为了实现这一目标，我们的目标是：1)分析监测数据并传播研究 从高优先领域通过同行评议的出版物和演示文稿面向目标受众；以及2) 与疾病预防控制中心合作，并在研究项目上授予受赠者。在我们计划的研究活动中， 检查MD患者的怀孕情况，评估参与临床试验和非临床试验的情况 参与，评估临床试验参与对MD自然病史的影响，并描述 心肺转归和治疗。这些活动产生的结果将向公众提供信息 MD对健康的影响，有助于临床了解MD的自然病史、生存和 死亡率，以及受MD影响的个人和家庭的医疗需求。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 提供服务，并将个人与MD及其家人联系起来，提供社会服务，以提高生活质量。