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年的肌肉营养不良社区援助，研究和教育修正案已授权 疾病控制与预防中心创建国家肌肉营养不良的流行病学 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，旨在进行基于人群的监视和长期随访 被诊断出患有Duchenne和Becker肌肉营养不良的人（DBMD）。 2011年，MD Starnet 扩展到包括另外七个MDS的横截面监视（先天性，远端，Emery- dreifuss，facioscapulohumeral，肢体女孩，myotonic和obulophalylngeal），在2014年，这扩展了 继续监视。我们的爱荷华州MD Starnet Core网站是MD的四个宪章奖之一 Starnet于2002年，并于2006年，2011年和2014年成功退还。作为MD Starnet Core网站，我们 通过整合，成功地对DBMD进行了基于人群的监视和额外的MD 爱荷华州主要合作伙伴提供的临床，程序化和推荐资源。我们与 爱荷华大学的生殖分子流行病学研究与教育计划 提供了经验丰富的研究人员来分析MD Starnet数据并在同行评审的手稿上发布 优先级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 扩展和扩展我们在分析和传播基于人群的监视和 九个MDS的调查数据。我们建议继续与CDC调查人员和 其他奖励的祖母，以传播专注于高优先级主题领域的研究并告知 知识的差距。为此，我们的目标是：1）分析监视数据并传播研究 从高优先级领域到经过同行评审的出版物和演示，到目标受众；和2） 与CDC合作，并授予Grandtees研究项目。在我们计划的研究活动中 正在检查MD患者的怀孕，评估临床试验参与和非 - 参与，评估临床试验参与对MD自然历史的影响，并描述 心肺结局和治疗。这些活动产生的发现将为公众提供信息 MD的健康影响，并有助于对自然历史，生存和 由MD影响的个人和家庭的死亡率以及医疗保健需求。这些发现也将指导 制定和提供政策和护理计划，以确保获得全面的医疗保健 服务并将个人与医学博士及其家人联系到将改善生活质量的社会服务。