Component A: Iowa MD STARnet Core Site

组件 A：爱荷华州 MD STARnet 核心站点

• 批准号: 10662427
• 负责人: Katherine Dianne Mathews
• 金额: $ 41.5万
• 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/10662427
• 关键词: Component; Iowa; MD; STARnet; Core

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 integrated clinical, programmatic, and referral resources provided by key partners in Iowa, such as the Iowa Neuromuscular Program and the Iowa Muscular Dystrophy Association, with the active, population-based resources of the Iowa Registry for Congenital and Inherited Disorders to conduct longitudinal surveillance of DBMD and cross-sectional surveillance for the seven additional MDs. Our partnership with the Reproductive Molecular Epidemiology Research and Education Program at The University of Iowa has provided experienced investigators to conduct data analyses and publish MD STARnet data. For the proposed project, we will use our highly skilled and successful team to extend and expand our leadership role in population-based longitudinal surveillance and research for the nine MDs. We propose to continue working collaboratively with CDC investigators and other grant awardees to implement standardized methods and data collection activities. To accomplish this, we aim to: 1) build new and maintain existing relationships with MD partners to enhance and promote surveillance efforts; 2) continue to conduct longitudinal, population-based surveillance using existing MD STARnet methods to identify and gather data on individuals with one of the nine MDs; and 3) develop research methods and tools to augment surveillance data collection. Among our planned research activities will be examining pregnancy among individuals with MD, evaluating clinical trial participation and non-participation, and assessing the impact of clinical trial participation on the natural history of MD. Findings generated from our proposed surveillance and research activities will identify 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 public health agencies in the development and provision of policies and programs that will 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 爱荷华州主要合作伙伴提供的成功整合临床，程序化和推荐资源， 作为爱荷华州神经肌肉计划和爱荷华州的肌肉营养不良症，与主动 爱荷华州先天性和遗传疾病的基于人群的资源 DBMD和横截面监视的纵向监视，对另外七个MDS进行了监视。我们的 与生殖分子流行病学研究与教育计划的合作伙伴关系 爱荷华大学提供了经验丰富的研究人员来进行数据分析并发布MD Starnet 数据。对于拟议的项目，我们将使用我们高技能和成功的团队来扩展和扩展我们的 在基于人群的纵向监视和九个MDS研究中的领导作用。我们建议 继续与CDC调查人员和其他赠款奖合作，以实施标准化 方法和数据收集活动。为此，我们的目标是：1）建立新的并维护现有 与MD合作伙伴的关系以加强和促进监视工作； 2）继续进行 使用现有MD Starnet方法识别和收集数据的纵向，基于人群的监视 关于九个MD的个人； 3）开发研究方法和工具以增加监视 数据收集。在我们计划的研究活动中，将检查患有 MD，评估临床试验参与和非参与的评估，并评估临床试验的影响 参与医学博士的自然历史。我们提出的监视和研究产生的发现 活动将确定医学博士的公共卫生影响，并有助于对自然的临床理解 受MD影响的个人和家庭的历史，生存和死亡率以及医疗保健需求。这些 调查结果还将指导公共卫生机构制定和提供政策和计划 将确保获得全面的医疗服务，并将个人与MD及其家人联系起来 社会服务将改善生活质量。

项目成果

Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy.

预防性使用心脏药物来延迟杜氏肌营养不良症的左心室功能障碍。

• DOI: 10.1002/bdr2.2260
• 发表时间: 2024
• 期刊: Birth defects research
• 影响因子: 2.1
• 作者: Conway,KristinM;Thomas,Shiny;Ciafaloni,Emma;Khan,RabiaS;Mann,JoshuaR;Romitti,PaulA;Mathews,KatherineD;MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)
• 通讯作者: MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)