Updated ‘Action Plan for Muscular Dystrophies’ Reflects Latest in Research and Patient Care

The Muscular Dystrophy Coordinating Committee (MDCC) has released an updated version of its Action Plan for the Muscular Dystrophies, a document that works as a comprehensive guide to address the challenges experienced by people with muscular dystrophy. The committee, authorized by Congress, is composed of representatives from U.S. federal agencies and patient advocates.

An overview of the plan and recommendations for its use were published as an editorial in the Muscle & Nerve journal.

MDCC’s Action Plan offers a review of the problems and obstacles faced by people who live with a chronic and genetic condition that affects the body’s capacity to produce proteins needed to form healthy muscle, resulting in progressive weakness and loss of muscle mass. The revised plan, first issued in 2005, was published as a result of research advances and new scientific methodologies, including in-depth genetic analyses.

“Funding organizations and other resource providers, scientists, patients and advocates can all use this plan in their own ways to guide research, collaborations and strategies to extend and improve the quality of life of people suffering from these disorders,” Stephen I. Katz, MD, PhD, the editorial’s senior author and MDCC chairman, said in a news release. Dr. Katz  also serves as director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases at the National Institutes of Health.

The plan also notes similarities between different types of muscular dystrophy, and offers patient perspectives on goals. It has 81 objectives organized in six different sections: understanding causes of muscular dystrophies; screening and diagnosis; developing treatments; preparing for clinical trials; providing care, management and access to services; and investing in research infrastructure and the workforce.

MDCC decided an update was necessary because of advancements in knowledge about the mechanism that cause muscular dystrophies, and in understanding of disease symptoms. In addition, since 2005 new clinical trials have been initiated and clinical management improved, enhancing  patients’ overall health and life expectancy.

“The action plan represents current thinking among experts in the field about what questions need to be answered and which obstacles need to be overcome so that we can accelerate progress in muscular dystrophy research, patient care and services,” said Glen Nuckolls, PhD, executive secretary of the MDCC and program director at the NIH’s National Institute of Neurological Disorders and Stroke.

There currently is no cure for the more than 30 diseases included in the group classified as muscular dystrophies, or effective treatments able to stop or reverse these disorders. Given the great unmet needs associated with these diseases, the MDCC was established by the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 (MD-CARE Act, Public Law 107-84)to coordinate efforts among patient advocacy groups, federal agencies, and researchers.

The complete Action Plan for the Muscular Dystrophies is available here.