Muscular Dystrophy Coordinating Committee Meeting

April 11, 2023
Hybrid | 1- 5pm EDT

Muscular Dystrophy Coordinating Committee Meeting - 04/11/2023
Time Presentation

Introductions and Meeting information

Glen Nuckolls, National Institute of Neurological Disorders and Stroke (NINDS/NIH), MDCC Designated Federal Official (DFO)


Chair Presentation

Diana Bianchi, Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD/NIH), MDCC Chair


Group Discussion



Meetings/Conference Report

Muscular Dystrophy Association Clinical and Scientific Conference 2023
Sharon Hesterlee, Chief Research Officer, MDA




NIH Proposal for Renewal of the Action Plan

Glen Nuckolls, NIH                                                                                                                                                                       


Group discussion and consideration of proposed renewal plan




Strategic Plans of MDCC Member Organization 

(This session will be continued for the Fall 2023 MDCC meeting)

  • Jamshid Arjomand, Chief Scientific Officer, FSHD Society
  • Tanya Stevenson, Chief Executive Officer, Myotonic Dystrophy Foundation
  • Marielena Mcguire, Program Manager, Congressionally Directed Medical Research Program United States Army Medical Research and Development Command
4:15-4:50 Discussion of integrating other dystrophy strategic plans into the Action Plan renewal
4:50 Closing Remarks
Diana Bianchi, NICHD/NIH
5:00 Adjourn

Action Plan Renewal Proposal

Glen Nuckolls, PhD (NINDS) explained that the Action Plan for the Muscular Dystrophies (MDs) was developed in 2005 and is due to be renewed in 2025. This renewal should represent the current views of a variety of stakeholders on strategies for advancing research, accelerating progress toward effective treatments, and enhancing the quality of life (QoL) of individuals living with MDs. Dr. Nuckolls proposed five sections for the 2025 Action Plan Renewal: (1) unique and common mechanisms of MDs; (2) preclinical therapy development; (3) clinical studies, including trial readiness, epidemiology, natural history, and clinical trials; (4) enhancing QoL and access to care and services; and (5) opportunities for collaboration and partnerships, including team science, data sharing, and public/private partnerships (PPP). Each section will have a dedicated working group, which will be managed by an MDCC Action Plan Steering Group that reports to the MDCC.

Strategic Plans of Other Organizations

The MDCC heard presentations from three organizations on their strategic plans. Priorities for the Facio-Scapulo-Humeral Muscular Dystrophy (FHMD) Assessment & Clinical Tools (FACT) Alliance include development of imaging biomarkers, pediatric natural history studies, remote and real-world assessments, innovation in trial designs and platform trials, and expansion of the Clinical Trial Network already managed by FACT. The Duchenne Muscular Dystrophy Research Program (DMDRP) has three strategic priorities: (1) accelerating discovery and development of therapeutics with a path into clinical applications, (2) advancing understanding of the effect of DMD on multiple body systems, and (3) expanding the DMD researcher community. The Myotonic Dystrophy Foundation has four overarching goals in its 3-year strategic plan, for which it is currently in Year 2. These goals are to (1) strengthen the myotonic dystrophy community, (2) improve and expand access to healthcare and resources to better meet the needs of individuals and their families, (3) eliminate barriers to accelerating drug development, and (4) build a strong and sustainable organization.

Presentation from the Chair

Dr. Bianchi, MD (NICHD) highlighted planned changes to increase public accessibility of findings from NIH-funded studies and to simplify review of research proposals. The three factors proposed for grant review are importance of the research, feasibility and rigor, and expertise and resources. She described future plans to support NIH’s Undiagnosed Diseases Network; the current program will expire in July 2023, but several Institutes and Centers are working to develop a new Network. She also described the FY23-FY27 NIH-wide Strategic Plan for diversity, equity, inclusion, and accessibility, as well as report findings from the Subgroup on Individuals with Disabilities. Dr. Bianchi briefly described the Advanced Research Projects Agency for Health (ARPA-H) program. Finally, she provided a brief overview of recently published NIH-funded research into the MDs.

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