News Release

Thursday, April 7, 2016

Publication highlights release of muscular dystrophy action plan

Plan provides strategies and opportunities for muscular dystrophy research.

The Muscular Dystrophy Coordinating Committee (MDCC), a congressionally authorized group of representatives from federal agencies and patient advocates, recently released an updated version of its Action Plan for the Muscular Dystrophies. The plan is a comprehensive guide for addressing critical challenges facing people living with muscular dystrophy. An editorial, published in Muscle & Nerve, provides an overview of the plan, as well as recommendations for its use.

“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,” said Stephen I. Katz, M.D., Ph.D., chair of the MDCC, director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases at the National Institutes of Health, and senior author on the editorial.

The updated plan reflects research advances and new scientific methodologies such as in-depth genetic analyses, emphasizes similarities among different forms of muscular dystrophy, and includes patient perspectives in a number of objectives.

The first MDCC Action Plan for the Muscular Dystrophies was issued in 2005. Since then, the field has made tremendous progress in understanding the mechanisms that cause muscular dystrophies and their symptoms, initiated clinical trials of potential therapies, improved clinical management, enhanced overall health, and lengthened life for people who have muscular dystrophies.

“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, Ph.D., program director at the NIH’s National Institute of Neurological Disorders and Stroke and executive secretary of the MDCC.

The 81 objectives of the 2015 action plan, first released in November 2015, are organized under six sections:

  • Understanding causes of the muscular dystrophies
  • Screening and diagnosis
  • Developing treatments
  • Preparing for clinical trials
  • Providing care, management and access to services
  • Investing in research infrastructure and the workforce

The muscular dystrophies are a group of more than 30 genetic disorders characterized by weakness and breakdown of muscles. There are no treatments available that stop or reverse these disorders; many are fatal.

The MDCC  was established by the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 (MD-CARE Act, Public Law 107-84) as a means to coordinate efforts among patient advocacy groups, federal agencies and researchers.

The 2015 Action Plan, along with lay-language highlights, can be found at

To arrange an interview with Dr. Stephen Katz, please contact Trish Reynolds,, 301.496.8190.

The NINDS is the nation’s leading funder of research on the brain and nervous system. The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

The mission of the NIAMS is to support research into the causes, treatment and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit

NIH…Turning Discovery Into Health®


Rieff HI et al. The Muscular Dystrophy Coordinating Committee Action Plan for the Muscular Dystrophies. Muscle & Nerve. doi: 10.1002/mus.25111