The Muscular Dystrophy surveillance, racking, and Research Network (MD STARnet) was established in 2002 to conduct population-based surveillance and long-term follow-up of cases with Duchenne and Becker muscular dystrophies (DBMD); longitudinal surveillance for DBMD has been ongoing since 2004. In 2011, the MD STARnet was expanded to include cross- sectional surveillance for seven additional MDs (congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal). Our Iowa Site was one of four charter awardees for the MD STARnet in 2002 and was refunded in 2006 and 2011. As an MD STARnet site, we have successfully integrated clinical, programmatic, and referral resources provided by key partners in Iowa, such as the Iowa Neuromuscular Program, with the active, population-based resources of the Iowa Registry for Congenital and Inherited Disorders to conduct longitudinal surveillance of DBMD and cross-sectional surveillance for the additional MDs. Also, 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. Overall, our infrastructure in Iowa now serves to advance knowledge about the prevalence of MD, clinical care for and co-morbidities diagnosed among MD cases, and the influence of family characteristics and social support on clinical outcomes. We propose to use our highly skilled and successful team to expand surveillance and research activities for MDs and to include additional, selected neuromuscular diseases (NMDs), specifically congenital myopathies, spinal muscle atrophy, Pompe disease and Charcot-Marie- Tooth disease. To accomplish this, we aim to: 1) build new and maintain existing relationships with MD and other NMD partners to enhance and promote surveillance efforts; 2) conduct longitudinal, population-based surveillance and research for all MDs by reviewing medical records and accessing administrative data; 3) pilot longitudinal surveillance for additional, selected NMDs; 4) develop and implement a communications plan to disseminate surveillance and research results to partners, patients, and policy makers to improve access to services and care; and 5) collaborate with CDC and other grantees to develop and implement an evaluation plan for the multi-site surveillance system. Using our proposed, population-based approach, we can gain increased insights into the epidemiology of MD and these additional NMDs and their complications. The data collected can also aid in providing treatment and support options beneficial to affected individuals and their families.
The Iowa Site will continue its leadership role in longitudinal surveillance and research for muscular dystrophies (MDs) and expand such activities to include additional, selected neuromuscular diseases (NMDs). Data collected can aid in providing treatment and support options and identifying unique needs of individuals with MDs and NMDs, as well as their families. Findings will be shared with scientists, clinicians, advocacy groups, an policy makers developing interventions and patient resources.
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