April 6, 2020 / Advocacy

91 Members of Congress Send Letter of Strong Support for Duchenne Priorities & Funding for Next Year’s Budget

As a result of 123 meetings with Members of Congress in Washington DC, and over 2,500 action alert emails sent by this community, we are pleased to report a very successful outcome for the Duchenne Fiscal Year 2021 (FY21) funding letters signatures.

91 members of Congress in total (68 in the House and 23 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 really important for these efforts. And this is an increase in signatures from last year!

PPMD is grateful to the Foundation to Eradicate Duchenne for their partnership and collaboration on the Advocacy Conference.


Centers for Disease Control

  • Asks Congress to increase funding for CDC’s Muscular Dystrophy Program to $8 million.
  • With that funding, evaluate the impact of the Care Considerations on patient outcomes, particularly in rural and underserved areas.
  • Evaluate the impact of the presence of a Certified Duchenne Care Center on patient outcomes.
  • Assess diagnostic odyssey and provider resource needs before and after implementation of recommendations made by the National Task Force for Early Identification of Childhood.

National Institutes of Health

  • Requests the NIH convene a multi-stakeholder workshop to evaluate pre-clinical models us to consider whether alternative models or strategies may improve therapy development outcomes.
  • Encourages the National Institute of Neurological Disorders and Stroke (NINDS) to develop a centralized resource to store and access de-identified data for further research.

Food and Drug Administration

  • Encourages the FDA expand on the implementation of 21st Century Cures to include in the benefit-risk framework a description of how patient experience data was considered.

Department of Defense

  • Support level funding of $10 million for the Duchenne Muscular Dystrophy Research Program (DMDRP) within the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP).
Download the letter


The timing for when the funding bill (known as appropriations) is finalized can often be unpredictable, particularly with so much happening with COVID-19, 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.


While on the Hill, advocates were also pushing for passage of the BENEFIT Act. The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act was re-introduced by Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) as S.3385 and was crafted in partnership with PPMD. The BENEFIT Act amends food and drug law to ensure that invaluable patient perspectives – such as input from patient preference studies, patient reported outcomes, and other types of patient experience data – are included as part of the FDA risk-benefit review of candidate new drugs. PPMD will send out additional alerts regarding this bill once Congress re-focuses its attention following COVID-19.


We appreciate that your time is precious, and we are grateful to everyone who made the trip to DC and to all those who advocated from home. This community is incredible and continues to lead the way in rare disease advocacy. We are so proud of the work you did and continue to do! We will be in touch with future action alerts related to BENEFIT.

Senate Signatures

Kyrsten SinemaAZ
Diane FeinsteinCA
Kamala HarrisCA
Michael BennetCO
Richard BlumenthalCT
Chris CoonsDE
Dick DurbinIL
Ed MarkeyMA
Elizabeth WarrenMA
Chris Van HollenMD
Angus KingME
Gary PetersMI
Debbie StabenowMI
Amy KlobucharMN
Roger WickerMS
Kevin CramerND
Jeanne ShaheenNH
Cory BookerNJ
Jacky RosenNV
Catherine MastoNV
Kirsten GillibrandNY
Sherrod BrownOH
Tammy BaldwinWI

House Signatures

Terri SewellAL07
Doris MatsuiCA
Mark DeSaulnierCA11
Jimmy PanettaCA20
Ted LieuCA33
Mark TakanoCA41
Joe NeguseCO02
John B. LarsonCT01
Jim HimesCT04
Jahana HayesCT05
Lisa Blunt RochesterDE01
Ted YohoFL03
Stephanie MurphyFL07
Bill PoseyFL08
Darren SotoFL09
Brian MastFL18
Ted DeutchFL22
Lucy McBathGA06
Danny K. DavisIL07
Raja KrishnamoorthiIL08
Jan SchakowskyIL09
André CarsonIN07
John YarmuthKY03
Cedric L. RichmondLA02
Jim McGovernMA02
Seth MoultonMA06
Ayanna PressleyMA07
Stephen F. LynchMA08
William R. KeatingMA09
Jamie RaskinMD08
Chellie PingreeME01
Jared Golden ME02
Elissa SlotkinMI08
Haley StevensMI11
Collin C. PetersonMN07
Wm. Lacy ClayMO01
Donald NorcrossNJ01
Andy KimNJ03
Josh Gottheimer

Tom MalinowskiNJ07
Albio SiresNJ08
Susie LeeNV03
Lee ZeldinNY01
Peter KingNY02
Kathleen RiceNY04
Jerold NadlerNY10
Max RoseNY11
Carolyn B. MaloneyNY12
Antonio R. DelgadoNY19
Anthony BrindisiNY22
John KatkoNY24
Brian HigginsNY26
Peter A. DeFazioOR04
Brian FitzpatrickPA01
Dwight EvansPA03
Mary Gay ScanlonPA05
Susan WildPA07
Glenn “GT” ThompsonPA15
Connor LambPA17
John RoseTN06
David KustoffTN08
Steve CohenTN09
Eddie Bernice JohnsonTX30
Colin AllredTX32
Ben McAdamsUT04
Abigail D. SpanbergerVA07
Peter WelchVT01
Adam SmithWA09

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