Component A: Iowa MD STARnet Core Site

点击翻译按钮获取中文摘要

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.