The muscular dystrophies re an extensive group of heterogeneous genetic neuromuscular conditions characterized by progressive muscle weakness and degeneration. The nine major types of muscular dystrophy (MD) vary by age of onset, muscle groups affected, genes involved, severity, and progression of disease. The Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) has provided a large amount of valuable data on Duchenne and Becker MD, but gaps still exist in our knowledge of Duchenne and Becker MDs in minority populations. We will establish a new Core MD surveillance project in North Carolina's Piedmont (central) region. More than 50% of the state's residents live in the Piedmont (5.3 million), of whom 24.7% are African American (approximately 1.3 million).
The specific aims are as follows:
Specific Aim 1 : We will establish and conduct population-based surveillance and longitudinal follow-up for MDs and other neuromuscular disorders in the Piedmont region of North Carolina. We will use the MD STARnet methodology.
Specific Aim 2 : Using data from Aim 1 and drawing on our experience analyzing data from MD STARnet, we will describe the prevalence and natural history of MD and other neuromuscular disorders in North Carolina, the health care service use and costs of NC residents with MD and other neuromuscular disorders, and disparities in their access to care. We will examine disparities by proximity to source of care, race, ethnicity, and SES, but examining racial disparities will be a high priority, as this project will be one of the first MD STARnet sites with substantial representation of African Americans. We will also examine the impact of specific clinical treatments on specific outcomes for specific types of MD and neuromuscular disorders.
Specific Aim 3 : Using our long-standing partnership with the University of North Carolina (UNC) and the North Carolina Department of Health, UNC's relationships with community neurologists, and new partnerships we will disseminate findings from MD STARnet within North Carolina in accordance with the Network's communications and dissemination plans.
Specific Aim 4 : In collaboration with CDC and other A &B grantees, we will (1) determine if methods and infrastructure for conducting surveillance for MDs can be used to conduct surveillance for spinal muscular atrophy or neuromuscular disorders proposed by other grantees, and (2) determine the most efficient and effective methods to conduct population-based surveillance for MD and neuromuscular disorders.
Specific Aim 5 : We will collaborate with CDC and other A &B grantees on Network tasks, including (1) modifying the MD STARnet protocol to incorporate linkage to administrative data, (2) determining other neuromuscular conditions for pilot surveillance, (3) developing communications and dissemination plans, (4) developing evaluation plan, and (5) participating in the steering committee.
The Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) collects critical information supporting research that aids improvements in diagnosis, care, and policy. North Carolina is culturally and racially diverse, and its inclusio in the MD STARnet would bring additional information on the prevalence and course of muscular dystrophy and other neuromuscular disorders among African Americans. We will build a MD STARnet surveillance system in the Piedmont region of North Carolina and, with key partnerships, analyze the data and disseminate information that supports a reduction in the age of diagnosis and provides critical data used to positively impact affected individuals and their families.