MD STARnet: Feasibility of Expansion to other Muscular Dystrophies

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DESCRIPTION (provided by the applicant): The muscular dystrophies are a group of inherited, disabling diseases for which there are no cures, only treatment of symptoms. Types of muscular dystrophy are distinguished clinically by age at onset and pattern of muscle group involvement. Among children, the most common types are Duchenne and Becker muscular dystrophies (DBMD), X-linked allelic disorders commonly reported to affect one in 3,500 male births. The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) was originally established to conduct population-based ascertainment and prevalence estimation for DBMD, as well as document early signs and symptoms, types of care received, and factors affecting outcomes of individuals with these disorders. In 2002, the Iowa Registry for Congenital and Inherited Disorders (IRCID) was one of four charter awardees for the MD STARnet and was successfully refunded in 2006. As an MD STARnet site, we have successfully integrated clinical, programmatic, and referral resources provided by relevant stakeholders in Iowa with the resources of the IRCID to establish a statewide infrastructure for DBMD surveillance and to implement long-term follow-up of individuals with DBMD and their families. The infrastructure created by the MD STARnet can now serve to advance knowledge about the prevalence, demographics, health care coverage, and clinical care received by individuals and families affected by other types of MD. For the current project, the IRCID proposes to engage existing stakeholders and establish partnerships with new stakeholders to successfully continue as a participating MD STARnet site to: 1) continue to conduct population-based surveillance and research for DBMD; 2) design and implement population-based surveillance for seven additional muscular dystrophies (congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal); 3) assess the methodology for conducting population-based surveillance for all muscular dystrophies; and 4) analyze data, publish articles, and disseminate reports using information collected for Duchenne Becker muscular dystrophies. Using the population-based approach of the MD STARnet, we can gain increased understanding of the epidemiology of these additional MDs and their complications. The information collected can also aid in providing treatment and support options beneficial to individuals with these additional MDs as well as identify unique needs of these individuals and their families. PUBLIC HEALTH RELEVANCE: The Iowa site of the Muscular Dystrophy Surveillance, Tracking, and Research Network will continue its leadership role in surveillance and research for Duchenne and Becker muscular dystrophies and expand its surveillance activities to include congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal muscular dystrophies. Information collected can aid in providing treatment and support options and identifying unique needs of individuals with these muscular dystrophies and their families. Findings will be shared with scientists and professionals developing interventions and patient resources.