Thanks to your advocacy at our annual spring Advocacy Conference supporting congressional funding of Duchenne research and care priorities, Congress has continued to fund federal agencies for this work and the President has signed off on the final legislation.
We thank our congressional champions, Senator Roger Wicker (R-MS), Senator Debbie Stabenow (D-MI), Representative Doris Matsui (D-CA), and Representative Peter King (R-NY) for their leadership to get this done.
The legislative package included funding for fiscal year 2021 spending (which included our priorities) as well as the COVID-relief package. As we requested, included in the fiscal year 2021 spending package:
- $6 million continued funding for Duchenne muscular dystrophy at the Centers for Disease Control and Prevention (CDC)
- $10 million continued funding for research through the Department of Defense Congressional Directed Medical Research Program
- Language from Congress encouraging/urging:
- CDC to consider how widely the Duchenne Muscular Dystrophy Care Considerations have been adopted across the country and whether this has led to changes in practice or an improvement in patient outcomes, particularly in rural and underserved areas, and the presence of a Certified Duchenne Care Center.
- CDC to assess diagnostic odyssey and provider resource needs before and after implementation of recommendations from the Mississippi Project conducted 2005-2008
- CDC to develop a plan to leverage the recently established diagnosis code for Duchenne to update MDSTARnet surveillance, expansion, and newborn screening efforts
- The National Institutes of Health (NIH) to significantly expand its support for research on Duchenne muscular dystrophy, particularly accelerating and optimizing the clinical trial process through novel and innovative trial designs, such as platform trials, which might serve as a model for other rare diseases communities
- NIH to convene a multi-stakeholder workshop to evaluate pre-clinical animal models used frequently in muscular dystrophy treatment research
- NIH to create a centralized resource to store and access de-identified data from National Institute of Child Health and Human Development’s (NICHD’s) supported studies
- The Food and Drug Administration (FDA) to build on patient-focus drug development (PFDD) work and assess the use of patient data in regulatory decision-making, which would be mandated by Congress in S. 3385, the BENEFIT Act, led by Sens. Wicker (R-MS) and Klobuchar (D-MN)
Join PPMD’s 2021 Virtual Advocacy Conference!
We look forward to our continued advocacy on these priorities next year for fiscal year 2022 spending. PPMD’s 2021 Advocacy Conference will be going virtual March 10, 2021. Be on the lookout for registration in the coming weeks.
We would like to offer our special thanks to Representative King, our long-time champion for Duchenne funding, as he retires from Congress at the end of this year. We are also very pleased to announce our new champion, Representative Steve Stivers (R-OH) who will be taking up the mantle with Representative Matsui to lead the appropriations request in the House next year. Our PPMD connections in Ohio run deep, with fantastic work being done at PPMD Certified Duchenne Care Centers located in Ohio, including Nationwide Children’s Hospital in Rep. Stivers’ district in Columbus, as well as Cincinnati Children’s Hospital and Akron Children’s Hospital. We look forward to working with all of our champions next year!Learn more about the Advocacy Conference >