Request for Information (RFI): MDCC Action Plan for the Muscular Dystrophies

Purpose

The MDCC previously developed and publicized the Action Plan for the Muscular Dystrophies (AP) as a strategic planning document of gaps and opportunities for research and strategies for improving the lives of affected individuals.  The AP was last renewed in 2015. In 2024, working groups composed of researchers, clinicians, patient advocacy group representatives and people with lived experience met to develop draft priorities for the 2025 renewal of the AP. We are now requesting comments from the public on these draft priority documents.  Input is requested from all stakeholders including people with lived experience, academic and industry researchers, clinicians, professional societies and non-profit organizations. 

View a video of the November 22, 2024 MDCC meeting including presentations from the AP working groups and discussions of their recommendations.

Anyone may comment on the draft priorities described here or provide suggestions for additional priorities. Comments/suggestions will be considered by the MDCC and its working groups for inclusion in the 2025 Action Plan for the Muscular Dystrophies (AP).

Background

The 2015 Action Plan for the Muscular Dystrophies(pdf, 690 KB) (AP) was developed with input from researchers, clinicians, non-profits, people with lived experience and government agencies.  The expert opinions in the plan have guided decision-making by researchers, clinicians, MDCC member organizations and others.  The 2015 AP contained a total of 81 objectives and goals in the categories of disease mechanisms, diagnosis/screening/biomarkers, preclinical therapy development, clinical therapy development, living with muscular dystrophy and infrastructure.  While significant progress has been made on these objectives, there is still much work to be done to advance knowledge of the muscular dystrophies, develop and implement effective therapies and enhance strategies to improve the lives of affected individuals.  While many of the 81 objectives of the 2015 AP remain important goals, the renewed 2025 version is being developed as a much smaller number of broad priorities to guide activities over the next ten years to advance muscular dystrophy research and improve accesses to care and services for affected individuals.

Guidance for submitting your ideas

The documents at this site describe draft priorities for the 2025 AP recommended by working groups of experts on the topics of 1) research for understanding the causes of muscular dystrophies (mechanisms of disease), 2) research for developing new treatments (preclinical translation), 3) clinical studies and standards of care, 4) thriving with muscular dystrophy (access to benefits, resources, diagnosis and clinical care), and 5) cross-cutting priorities that can accelerate progress in multiple areas. 

Please consider these draft priorities and provide your perspectives on the importance of these priorities; anything to add or modify and anything you would prioritize that is not already included. 

Please consider the following questions in your submitted responses: