On February 27, 2001, 20 years ago, the Duchenne community gathered in Washington, DC for the first ever Senate hearing focused on Duchenne muscular dystrophy.
On that much-anticipated day, a young teenager, Ben Cumbo, Goodwill Ambassador to the MDA, testified in front of a Senate committee and essentially said: “Senator Specter, the government can spend $2 billion on a plane, $100 million for muscular dystrophy over five years wouldn’t hurt.” He asked everyone to imagine what progress we would make if we too had the investment of just one stealth bomber. The audience response was immediate and thunderous.
Senator Specter asked Ben what he wanted and what was most important to him. Ben replied that he wanted every person diagnosed to have treatment, not just him… and he wanted to go to college, have a career in international policy, and a girlfriend.
As our community knows, sadly Ben is no longer with us. While we lost him way too soon, we are so proud of him for achieving a number of his goals, including earning a graduate degree in international studies and working in the profession of his choosing.
To all of us who sat in that Senate hearing room 20 years ago, the HOPE we felt was palpable. Our voices, our consistent message, the belief that a piece of legislation would and could change the world of Duchenne.
Later that year, Congress would enact the landmark Muscular Dystrophy Community Assistance, Research and Education Act (MD-CARE Act) which unleash a multi-faceted federal public health and medical research attack on Duchenne and other forms of muscular dystrophy. It would be followed by two updates to the law as well as other laws and policies that have helped transform the field over two decades.
2001 Senate Hearing
Fast forward 20 years
Your voices, each and every voice, reaching out to your representatives in the House and Senate with a consistent message – Invest in research, develop centers of excellence to stimulate ideas and collaborations, understand incidence (how many people are currently living with the Duchenne) and prevalence (how often it occurs) and develop Standards of Care to ensure optimal care for every individual – has enabled us to achieve much. And when our community arrived at the footsteps of the FDA, we were ready with a foundation that enabled FDA to heed our message of considering the perspective of the patient and caregiver and to exercise appropriate flexibility in their review and decision-making.
Over the years, PPMD advocates have made more than 2500 in-persons visits to Capitol Hill and countless more district visits and phone calls. You have told your own stories of living with Duchenne and Becker, including your own hopes and goals just like Ben did.
Long ago, when my sons, Chris and Patrick were first diagnosed, I believed Duchenne could and should have a voice. I believed then and still believe today that every single voice matters. Duchenne is not a political issue. It has been supported from elected officials on the right, left, and center. It is a human issue, a collective story about the voice of individuals with Duchenne and the people who love them, coming together around a common vision and plan to change the status quo.
This is a success story that continues to be written each passing day. The MD-Care Act and its follow-on laws have given us the Wellstone (research) centers, the MD STARnet (surveillance) program, care standards and a coordinated comprehensive research program. Other laws and actions have brought changes to the FDA that have helped deliver four FDA-approved therapies and more than 30 active clinical trials in Duchenne.
While Ben passed away in 2015, his wish has come true. We have a stealth bomber invested in Duchenne – and even more when you consider the significant amount of non-governmental dollars the federal funding has leveraged. But we have much more to still do to make Duchenne and Becker conditions that are widely treatable and manageable. Our commitment, our advocacy is passionate, relentless, unceasing and driven to end Duchenne.
This dream is built upon the shoulders of Ben and the many other advocates whom we have lost but who have helped get us to this point. As we spend time to commemorate what we have achieved over the past two decades, I am most excited about what I know we will accomplish over the next two and beyond. As we look to the work still to be done, we hope you will lend your voice to advocacy, for Ben, for Chris, for Patrick, for everyone who paved the way toward progress.
Join PPMD’s 20th Anniversary of Advocacy in Action
Virtual Town Hall on Facebook Live | February 3
To commemorate the passage of the MD-CARE Act, PPMD will host a Virtual Town Hall on Wednesday, February 3, 1 PM – 4 PM ET to look back on past accomplishments and progress made possible by Duchenne advocates, as well as looking ahead at work yet to be done in this new era.
The agenda will feature panel discussions by NIH, FDA, and CDC leadership, personal reflections from our Duchenne Senate and House Champions, as well as reflections from community members, families and friends who have been influential in our progress in Washington D.C.
You won’t want to miss this informative and inspiring event open for all to join!RSVP on Facebook >
2021 Advocacy Conference: Virtual Fly-In | March 10
Join us on Wednesday, March 10 for PPMD’s 2021 Advocacy Conference: Virtual Fly-In. While we might not be able to be face-to-face in Washington, DC this year, we are still able to make our voices heard.
Over the last 20 years, Duchenne Advocacy has resulted in…
- Over $600 million in funding for Duchenne related programs at the National Institutes of Health, Center for Disease Control, and Department of Defense.
- A standard of care established and recently updated for all those diagnosed with Duchenne, which has markedly improved the quality of life and extended lifespan.
- An ever growing Duchenne drug development pipeline with over 40 companies developing drugs for Duchenne.
There is no travel or financial expense associated with this virtual event. Only your time and energy are needed to join a movement and help keep Duchenne as a priority in our legislative environment.Register >