Surveillance of Muscular Dystrophies - The various forms of muscular dystrophy are individually rare, but these devastating neuromuscular disorders collectively have a major impact on the health and lives of affected individuals, family members, and communities. Such diseases have attracted greater attention from the public, health care providers, and pharmaceutical/biotechnology firms in recent years, but despite the higher profile, there remains a need for increased knowledge about the epidemiology, access to healthcare resources, and outcomes of these disorders across demographic groups in the United States. The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) began in the early 2000s to help illuminate some of the epidemiologic and health outcomes issues in muscular dystrophies. At its inception, the focus was on individuals with diagnoses of Duchenne and Becker muscular dystrophy. MD STARnet expanded in 2011 to include surveillance across sites for congenital (CMD), distal (DD), Emery-Dreifuss (EDMD), facioscapulohumeral (FSHD), limb-girdle (LGMD), myotonic (DM), and oculopharyngeal (OPMD) MDs. In 2019, the Florida MD STARnet site (23 counties in Northern Florida) became a new site for MD STARnet. As a new MD STARnet site, we successfully conducted population-based surveillance for the 9 inclusive MDs through coordinated clinical resources and collaborations with key state health authorities in Florida. For the proposed project, we will continue to use MD STARnet methodology to analyze and disseminate population-based surveillance for 8 MDs (BMD, CMD, DD, DMD, EDMD, FSHD, LGMD, DM) through our experienced investigators, on-going collaborations with the Florida Department of Health, and expanded hospital partnerships. We will continue to work collaboratively with CDC and other MD STARnet sites to disseminate research that focuses on high priority topic areas. Our aims are to 1) Conduct rigorous population-based muscular dystrophy surveillance and data analysis, 2) Conduct data analyses on high-priority and contemporary areas of research in collaboration with CDC and other MD STARnet grantees; and 3) Disseminate findings of analyses through presentations to target audiences and peer-reviewed publications. The results of these studies will help with health policy decision-making and provide accurate epidemiologic information on muscular dystrophies that will be helpful for health care providers to know.
