This year marks 20 years since passage of the MD-CARE Act, landmark legislation led by PPMD that ushered in a new era of federal investment into the muscular dystrophies. This year also marked the first time ever PPMD organized an all-virtual Advocacy Conference.
It was a record-breaking year for participation, with over 425 advocates participating from 43 states in over 300 virtual meetings. We also had over 1,000 action alerts sent to Congress from community members across the country while advocates participated in meetings.
Record breaking participation has translated into record breaking results! We are pleased to report a very successful outcome for the Duchenne Fiscal Year 2022 (FY22) funding letters signatures.
122 members of Congress in total (94 in the House and 28 in the Senate) agreed to support our request sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. Both letters have bi-partisan support, which is critical for these efforts.
PPMD is grateful to the Foundation to Eradicate Duchenne for their partnership and collaboration on the Advocacy Conference.
WHAT WAS IN THE REQUEST TO CONGRESS?
CENTERS FOR DISEASE CONTROL
- Asks Congress to increase funding for CDC’s Muscular Dystrophy Program to $8 million and that money be used to evaluate:
- The impact of the Duchenne Care Considerations, including on rural & underserved areas.
- Availability of consistent and coordinated care for adults with Duchenne.
- Impact of Duchenne and Becker on mental health of patients and caregivers.
- It also requests that CDC issue a report on program priorities including progress to date and priorities for upcoming years.
NATIONAL INSTITUTES OF HEALTH
- Requests the NIH to focus Duchenne research on:
- Challenges related to gene therapies, including delivery to individuals with neutralizing antibodies, manufacturing, and redosing of gene therapy.
- The impact of Duchenne on both the brain and the heart.
FOOD AND DRUG ADMINISTRATION
- Encourages the FDA to update the 2018 FDA Guidance on Duchenne to reflect advancement in gene therapies.
- Convene a meeting to evaluate how to minimize the use of placebo in clinical trials.
DEPARTMENT OF DEFENSE
- Requests an increase from $10 million $12 million for the Duchenne Muscular Dystrophy Research Program (DMDRP) within the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP).
|House Member||State and District|
|Terri A. Sewell||AL-7|
|Nanette Diaz Barragán||CA-44|
|Ami Bera, M.D.||CA-7|
|John B. Larson||CT-1|
|Eleanor Holmes Norton||DC at large|
|Lisa Blunt Rochester||DE|
|Gus M. Bilirakis||FL-12|
|Alcee L. Hastings||FL-20|
|Danny K. Davis||IL-7|
|James P. McGovern||MA-2|
|Stephen F. Lynch||MA-8|
|Daniel T. Kildee||MI-5|
|Emanuel Cleaver, II||MO-5|
|Donald M. Payne, Jr.||NJ-10|
|Bill Pascrell, Jr.||NJ-9|
|Carolyn B. Maloney||NY-12|
|Andrew R. Garbarino||NY-2|
|Elise M. Stefanik||NY-21|
|Joseph D. Morelle||NY-25|
|Thomas R. Suozzi||NY-3|
|Kathleen M. Rice||NY-4|
|Peter A. DeFazio||OR-4|
|Mary Gay Scanlon||PA-5|
|Sheila Jackson Lee||TX-18|
|Eddie Bernice Johnson||TX-30|
|Gerald E. Connolly||VA-11|
|Abigail D. Spanberger||VA-7|
|Peter Welch||VT at large|
|Carol D. Miller||WV-3|
|Member First||Member Last||State|
WHAT HAPPENS NEXT?
The timing for when the funding bill (known as appropriations) is finalized can often be unpredictable and can be delayed for many reasons – rest assured, they eventually get done. The good news is you have done the hard part already, ensuring the best chance for Duchenne related report language and funding are included for agencies that impact Duchenne in the coming year. We will keep you posted when the bills pass through and are signed into law.
Thank you for all your efforts!
Save the Date for next year’s in-person Advocacy Conference
The 2022 Advocacy Conference is planned to be in person next year March 6-8, 2022. We hope to see you in Washington D.C.! Sign up for PPMD emails to be notified when registration is open.