Surveillance of Muscular Dystrophies Component A: Iowa MD STARnet Core Site - In 2002, the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) was established in response to the Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 authorizing the Centers for Disease Control and Prevention to create a National Muscular Dystrophy Epidemiology Program. The mission of MD STARnet was to conduct population-based surveillance and long-term follow-up of individuals diagnosed with Duchenne and Becker muscular dystrophies (DBMD). In 2011, MD STARnet expanded to include cross-sectional surveillance for seven additional MDs (congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal), and with funding cycles in 2014 and 2019, this expanded surveillance has been continued. Our Iowa MD STARnet core site, a charter awardee for MD STARnet in 2002, has been successfully refunded in 2006, 2011, 2014, and 2019. 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, with the active, population-based surveillance resources of the Iowa Registry for Congenital and Inherited Disorders to conduct longitudinal surveillance of MDs. In addition, 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. Our proposed project for RFA-DD-24-0064, will continue to use our highly skilled and successful project team to extend and expand our leadership role in population-based longitudinal surveillance for the eight eligible MDs. We propose to continue working collaboratively with CDC investigators and other funded sites to implement standardized methods and data collection activities. To accomplish this, we will: 1) conduct longitudinal, population-based surveillance using existing MD STARnet methods to identify and gather data on individuals diagnosed with one of the eight eligible MDs; 2) implement the standardized MD STARnet database and applications for data collection and reporting; and 3) disseminate surveillance results to healthcare providers, partners, policymakers, and families. Among our planned studies for DBMD will be to improve understanding and inclusion in clinical trials for individuals with DBMD and to examine the age at which vital capacity falls below 1 L and if it is prognostic of disease progression and death. In addition, we propose to continue and expand our studies of pain experienced by people with MD and their use of prescription pain medication. Findings generated from our proposed surveillance and studies 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.
