Surveillance and Research of Muscular Dystrophies and Neuromuscular Disorders

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• Purpose. Using methods based on those from the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet), this funding opportunity announcement (FOA) will support: 1. Conducting population-based surveillance, longitudinal follow-up, and public health research for muscular dystrophies and neuromuscular disorder(s): 1) to develop estimates for prevalence, survival, healthcare service use and costs, disparities in access to care (by proximity to source of care, race/ethnicity, and SES), and 2) to determine associations between specific clinical treatments and specific outcomes for specific types of MD and neuromuscular disorders, and 3) to support public health and clinical practice for prevention of morbidities and their progression among persons with muscular dystrophy and neuromuscular disorders 2. Expanding the number of surveillance site(s) to increase the demographic, racial, and ethnic diversity in the current MD surveillance system 3. Sharing data with stakeholders seeking to improve health and access to care of people living with these conditions 4. Evaluating, modifying, and documenting the methodology and infrastructure of the surveillance system 5. Conducting pilot studies that investigate specific MD knowledge gaps and sharing results with participants and stakeholders This FOA has four components to achieve the purpose of the program. Components A and B consist of the core activities. Component A - core (existing MD surveillance and research programs) consists of using methods based on those from MD STARnet and lessons learned from previous MD surveillance systems to conduct surveillance and longitudinal follow-up for muscular dystrophies and neuromuscular disorder(s); maintaining current and building new key partnerships; analyzing data to produce findings useful for public health practice; disseminating results to stakeholders using data collected from current and previous MD surveillance programs; and evaluating, modifying, and documenting the surveillance methods and infrastructure. Component B - core (new MD surveillance and research programs) consists of building a system using methods based on those from MD STARnet to conduct surveillance and longitudinal follow-up for muscular dystrophies and neuromuscular disorder(s) in a population with a large minority group (i.e. African Americans, Hispanics, Asians); building new key partnerships; analyzing data to produce findings useful for public health practice; disseminating data to stakeholders; and evaluating, modifying, and documenting the surveillance methods and infrastructure. Component C consists of the data coordinating center (DCC) and abstractor training and quality assurance/control that support the core activities. Component D consists of additional muscular dystrophy research studies in which applicants design a study to address a knowledge gap in MD and share data with study participants and MD stakeholders. (Note: The methodology for component D studies will not be known until applications have been submitted. Studies that collect data on 10 or more people are required to have OMB approval unless one of several exemptions apply. Data will not be collected without the Office of Management and Budget’s (OMB) approval.)

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