The various forms of muscular dystrophy are individually rare, but these are devastating neuromuscular disorders that 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, knowledge about the epidemiology, access to healthcare resources, and outcomes of these disorders remains scarce, especially for members of minority populations. It is particularly difficult to study these issues for muscular dystrophy given the complex array of genetic subtypes that have been delineated for this category of inherited muscle disease over the past several decades. To help address these knowledge gaps, we proposed to establish a new site for the MD STARNet consortium, focusing on a discrete geographic region of northern Florida that has a substantial population base. This population-based study will be facilitated by a collaboration with the Florida Department of Health and the vast databases available to the Institute for Child Health Policy at the University of Florida. This project will yield new, important knowledge regarding the epidemiology, access to care, and outcomes of muscular dystrophy. Given the sizeable minority populations that exist in the areas to be studied, it is expected that much new knowledge regarding these populations will be derived from the proposed work.
This proposal involves a study of the epidemiology, access to care, and health outcomes for nine different subtypes of muscular dystrophy in a discrete geographic region in northern Florida. This region encompasses the more densely populated counties in northern Florida, providing an excellent opportunity to study muscular dystrophy in a region where this has not in the past been extensively examined. The methods used will be ones established by MD STARnet, a national consortium of investigators who have been examining these questions in various locations across the United States. The results of these studies will help with health policy decision-making, and also provide accurate information that may be used by health care providers in Florida and beyond.