Request for Information (RFI) on the Action Plan for the Muscular Dystrophies

Notice Number: NOT-NS-14-039

Key Dates
Release Date: August 30, 2013
Response Date: December 20, 2013

Related Announcements

Issued by
National Institute of Neurological Disorders and Stroke (NINDS)
National Heart, Lung, and Blood Institute (NHLBI)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)


The NIH, together with the Muscular Dystrophy Coordinating Committee (MDCC), is leading an effort to update the Action Plan for the Muscular Dystrophies, which was released in 2005. To help inform this planning process, the NIH and the MDCC welcome comments from the public regarding accomplishments since the Plan’s approval, remaining opportunities, and suggestions for new research objectives.


NIH’s mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability. The National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and the National Heart, Lung, and Blood Institute (NHLBI) fund the majority of muscular dystrophy-related research at NIH and work together to coordinate muscular dystrophy research and activities across NIH.

The muscular dystrophies are a group of hereditary, progressive degenerative disorders causing weakness of the skeletal or voluntary muscles. There are many different forms of muscular dystrophy, which differ in their age of onset, severity, and pattern of muscles and other body systems affected.

Among its provisions, the Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) established the MDCC and prompted a series of activities leading to the development of the Action Plan for the Muscular Dystrophies. The Action Plan, developed with input from an MDCC Scientific Working Group, includes research objectives in five areas: Mechanisms of Muscular Dystrophy, Diagnosis and Screening of Muscular Dystrophy, Therapy of Muscular Dystrophy, Living with Muscular Dystrophy, and Research Infrastructure Needs for Muscular Dystrophy.

Through the individual and collaborative activities of researchers, clinicians, and stakeholders in the muscular dystrophy community, driven, in part, by the Action Plan, there have been significant advances in understanding of disease mechanisms, development of new diagnostic tools that reduce the diagnostic odyssey for affected individuals and their families, evolution of drug discovery and development partnerships that have advanced candidate therapeutics, and improvements in the care and quality of life for affected individuals. While each type of muscular dystrophy is distinctive, and has required disease-appropriate activities, each has benefited from a shared focus of the muscular dystrophy community. At this time, there are new challenges and gaps that require an assessment of progress and a re-focus of current needs.

Information Requested

The NIH and the MDCC invite the muscular dystrophy research, advocacy, and family communities to provide comments from their perspective on progress made toward the objectives in the Action Plan, and on remaining opportunities. NIH also welcomes suggestions for modifications to the existing research objectives, research objectives that are no longer needed, and new objectives that should be considered for the updated Action Plan. The collected comments will be reviewed and considered as the MDCC and an MDCC Scientific Working Group develop the updated Action Plan.

How to Submit a Response

Responses to this RFI must be submitted electronically using the web-based format at: Responses will be accepted until December 20, 2013.

Responses to this RFI are voluntary. Submitted information will not be considered confidential. Any personal identifiers will be removed when responses are compiled.

This request is for information and planning purposes only and should not be construed as a solicitation or as an obligation on the part of the Federal Government. The NIH does not intend to make any awards based on responses to this RFI or to otherwise pay for the preparation of any information submitted or for the Government's use of such information.

The NIH will use the information submitted in response to this RFI at its discretion and will not provide comments to any responder’s submission. However, responses to the RFI may be reflected in future funding opportunity announcements. The information provided will be analyzed and may appear in reports. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted.  No proprietary, classified, confidential, or sensitive information should be included in your response. The Government reserves the right to use any non-proprietary technical information in any resultant solicitation(s).


Please direct all inquiries to:

John D. Porter, Ph.D.
Executive Secretary, Muscular Dystrophy Coordinating Committee
National Institute of Neurological Disorders and Stroke
Telephone: 301-496-5745