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. FSH Society played a key role in the formation of the MDCC through its sustained advocacy work with the federal government, and Daniel Perez, President and CEO of the Society, serves on the MDCC as a voice for the FSHD community.
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. A press release from National Institutes of Health describes the Action Plan as follows:
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 2015 Action Plan, along with lay-language highlights, can be found at http://mdcc.nih.gov/action_plan.
Reference: Rieff HI et al. The Muscular Dystrophy Coordinating Committee Action Plan for the Muscular Dystrophies. Muscle & Nerve. doi: 10.1002/mus.25111
Dear Daniel Perez,
Do you know if Deflazacort also works on FSHD ???
Also, where I could obtain a used or discarded standing wheelchair ???
I live in NY C, and do not receive any help from Medicare, public assistance or MD Org.
Any information would be deeply appreciated, sincerely, manny.
Deflazacort has not been tested in FSHD patients. Its mechanism of action is similar to prednisone, which has not been shown to improve strength in a short (12-week) trial in FSHD patients (http://www.ncbi.nlm.nih.gov/pubmed/9008492). A French retrospective study suggested that prednisone may have adverse effects in FSHD patients.
To find a wheelchair, check out the Abilities Expo at the end of this month in NYC:
http://www.abilities.com/newyork/donate.html
The Abilities Expo would also be a good place to seek information to help you get the assistance you need. Good luck!