Component C: Iowa MD STARnet Site Data Dissemination

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 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.