Component A: Iowa MD STARnet Core Site

组件 A：爱荷华州 MD STARnet 核心站点

• 批准号: 10220774
• 负责人: Katherine Dianne Mathews
• 金额: $ 37.5万
• 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/10220774
• 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 成功整合了爱荷华州主要合作伙伴提供的临床、项目和转诊资源， 作为爱荷华州神经肌肉计划和爱荷华州肌肉营养不良协会，与积极的， 爱荷华州先天性和遗传性疾病登记处的基于人口的资源， DBMD的纵向监测和另外七个MD的横截面监测。我们 与生殖分子流行病学研究和教育计划的伙伴关系， 爱荷华州大学提供了经验丰富的调查人员进行数据分析并发布MD STARnet 数据对于拟议的项目，我们将利用我们的高技能和成功的团队，以扩大和扩大我们的 在九个医学博士的基于人群的纵向监测和研究中发挥领导作用。我们建议 继续与疾病预防控制中心的调查人员和其他赠款获奖者合作， 方法和数据收集活动。为了实现这一目标，我们的目标是：1）建立新的和保持现有的 与MD合作伙伴的关系，以加强和促进监测工作; 2）继续开展 使用现有MD STARnet方法进行纵向、基于人群的监测，以识别和收集数据 对患有九种医学博士之一的人进行研究;以及3）开发研究方法和工具，以加强监测 数据收集。在我们计划的研究活动中，将检查患有 医学博士，评估临床试验参与和不参与，并评估临床试验的影响 参与MD的自然史。从我们拟议的监测和研究中得出的结果 活动将确定MD的公共卫生影响，并有助于对自然疾病的临床理解。 病史、生存率和死亡率以及受MD影响的个人和家庭的医疗保健需求。这些 研究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保健服务，并将MD患者及其家人联系起来， 改善生活质量的社会服务。