I had no intention of being part of the Duchenne community this long.
When we started PPMD in 1994, I naively thought we would have a cure and I would be spending these years with my sons and daughters and a ton of grandkids I would spoil rotten.
And here we are 25 years later. My sons are no longer here, I worry about my daughters being carriers, I worry about my granddaughter being a carrier, and our community has never had to work harder and the losses have never felt more profound.
Once we dove in and began to understand Duchenne – while simultaneously educating those around us – we understood that progress wasn’t going to come in the form of a magic wand or silver bullet. With time, we have had to redefine what progress is. And while it may be too late for my boys and all of the children we have lost along the way, there is progress. With every victory, large or small, we honor our brave loved ones.
PPMD started with two basic ideas in mind:
- We must give parents a community to turn to and a voice to advocate for their child’s healthcare.
- We must End Duchenne.
Both of these goals have gone through their own redefinition.
More than a Parent Project
When my sons were diagnosed in 1984, I went to the library to find everything I could on Duchenne. This was before the internet. We’re talking microfiche and a card catalog! My husband and I are both medically trained – he’s a retired family doc and I was in nursing – but Duchenne was something we maybe covered in a half-day course on neurological diseases if that. It was hard to find a community, to build an army to fight this horrible disease. We needed to unite all of the families that, like ours, felt alone.
The Duchenne community has never been stronger. It continues to grow and strengthen and is a force of nature in the international rare disease community. We have become a model for other diseases and a case study in how parent advocacy can unite and get things done.
We also realized that this isn’t just a ‘parents’ project.’ There are many voices joining us in this fight – entire families, whole communities, friends from work, our child’s school, not to mention regulatory agencies, pharmaceutical companies, biotechs, universities, and clinics.
But perhaps the most important voice we hear today is that of the people living with Duchenne. A voice that for years had been left out of the conversation and today is often leading the narrative.
Parent Project Muscular Dystrophy is a community-wide effort that we strive to help lead, incorporating the priorities and passion of every stakeholder.
Redefining an End to Duchenne
To me, success was going to be when we could turn off the lights at PPMD, close the door, and not look back. Because Duchenne would be done. Maybe it would be a vaccination you got as a child, a quick shot to guarantee you don’t ‘catch’ Duchenne, like the Polio vaccine. It would maybe take 5 years – 10 at the most, but only because nothing happens quickly with government agencies. That was my plan in 1994.
What we have learned is that Duchenne is complicated. I know, sounds simplistic and it almost feels ridiculous to write. But it’s true – if it was easy, PPMD wouldn’t be turning 25.
Duchenne is a progressive disorder that needs to be treated with a progressive attack plan. We didn’t realize in those early days that it was going to take a combination of therapies to treat Duchenne. We know that now. We also know that the fight won’t end when we have treatments. We have to make sure that our loved ones can access these treatments and that insurance companies will cover them.
In my 25 years at PPMD, I realize that this fight is an evolution: as our understanding of Duchenne evolves, so does our strategy to end it.
Every Day, Every Angle
Not only did we realize it would take a combination of therapies to treat Duchenne, we realized it would take a combination of approaches to fight Duchenne. PPMD is the only Duchenne-specific nonprofit in the United States that takes a truly comprehensive approach to address the full impact and progression of Duchenne.
We continue to invest in nearly all potential therapies, amplify every single voice as we advocate to both local and federal governments, and offer support and care for each family living with a diagnosis. For 25 years, PPMD has helped to change the landscape of Duchenne in research, advocacy, care, and engagement.
We will continue to relentlessly attack Duchenne for the next 25 years, or 50 years, whatever it takes, from every angle, using every weapon in our arsenal – the greatest of which is you.
PPMD has contributed to almost every Duchenne research strategy that is currently in the pipeline, including an initial investment in the very first Duchenne Muscular Dystrophy Research Center (DMDRC) led by Dr. Eric Hoffman. This investment in 1997 represented a shift away from the standard methods of doing research in isolated labs. Since that moment, the Duchenne research community (and rare diseases as a whole) would learn to work together to build ideas, to test theories, and to motivate each other. PPMD brought urgency and a willingness to take chances to the table. We broke down established barriers and pushed boundaries. We still do.
PPMD continues to drive change in the research arena. Our grant cycles offer opportunities to investigators and help us keep our finger on the pulse of research. PPMD’s Scientific Advisory Board expertly reviews every grant that comes in and offers funding or advice on how to proceed to garner funding in the future. Our collaboration with academic research institutes and industry partners, helps us push potential treatments to clinic faster, which in turn provides access to the Duchenne community and patients faster.
We don’t just look at one problem, or one bit of research, but rather we are looking comprehensively at the entire landscape and trying to intercept areas where time could be lost and opportunities missed.
Remaining idle is not an option for PPMD. When we see a gap we fill it. If we see a need, we address it. And where we see the path, we fearlessly and strategically work toward clearing it.
Like the creation of PPMD itself, our Advocacy program evolved from parents navigating their child’s diagnosis, frustrated at the limited federal support for research and services. Frustration turned to ambition and we headed to our nation’s capital, ready to speak up and demand that the federal government acknowledge the significant impact of muscular dystrophy on our families.
Each year, since 2000, dozens of families from across the U.S. descend upon Washington, D.C. for PPMD’s Annual Advocacy Conference. Over the course of two intense days, advocates visit the offices of hundreds of members to tell their story. People with Duchenne and their families use their voice to remind policy makers that they are contributing members of society and that Duchenne deserves the same federal priority and investment as any other disease.
As advocates, our most important role is to speak up on behalf of those who can’t. We must remind Congress that even when budgets are tight, funding Duchenne research is still a national priority. We must remind regulatory agencies that people with Duchenne and their families are willing to take risks if there is any potential benefit to come from an experimental treatment. And we must remind all policy makers and regulators that the clock is ticking.
We don’t have time for bureaucracy. Our children’s lives depend on it.
Perhaps the greatest advance in the fight to end Duchenne over the last several decades has been in the way we care for people diagnosed with Duchenne. In our 25 history, the average lifespan of people with Duchenne has increased from late teens/early-20s to mid-20s/early 30s. This incredible leap is because our community has pushed to advance care of the last quarter of a century.
When PPMD first began, there were no standards of care. Doctors and clinicians were left comparing notes, taking chances, and using a “wait and see” approach. Early in our history, PPMD insisted that steroids be considered as an early intervention and labeled as the “gold standard” due to their ability to slow the progression of muscle deterioration.
We take our leadership role in the Duchenne community very seriously and we know that people turn to us first when they have questions about research, advocacy, and most often, care. Over the last few years, PPMD has worked diligently with experts in our community to ensure that our website is up-to-date. We know that in urgent situations, families need important and accurate information at their fingertips. Not only does our website contain the critical information you need, but we introduced an Emergency Care Card and the first Duchenne-specific mobile app in the U.S. so you always have information you need when and where you need it. Our website provides comprehensive care handouts, separated by both stage of disease and body system that can be printed from home and taken to your medical team so that they have the latest Duchenne information.
For us, it’s always been about getting you the information you need to demand the care you deserve, from the best clinics available. Care is evolving and PPMD will continue to make sure we stay on top of critical information you need to know, when you need to know it.
Receiving the diagnosis of Duchenne is a devastating and isolating moment for a family. It is our goal to provide families with lifelines to the Duchenne community so that no one ever feels alone.
PPMD has always believed that knowledge is key in the fight to end Duchenne. Since our earliest days, PPMD has strived to not only continue to learn as much as possible about this ever-complex disorder, but then to share that with the Duchenne community.
To do so, PPMD hosts numerous in-person and virtual events throughout the year, including our Annual Conference, Annual Advocacy Conference, End Duchenne Tour, and educational webinar series. These events convene the leading experts in Duchenne research and care who provide updates on the most promising therapies in development and the most critical updates in care.
Participating in The Duchenne Registry is one of the most powerful ways you can stay updated on clinical trials and the latest advances in research. Plus, when you join the Registry, you are strengthening the power of a 10-year-old network of patient-powered data that will be used to improve care for people living with Duchenne and increase our understanding of the disorder. You become a citizen scientist by contributing to real scientific research, without ever leaving your home.
It is important to PPMD to be able to offer programs and actions that help empower families to move forward in the face of Duchenne, to unite with others in changing the landscape, and to be part of a community that is there for each other through good times and bad.
Not only do we host impactful in-person conferences and tour stops, we also provide local opportunities for families to gather. Whether through PPMD’s Connect program – our regional, parent-led outreach program – or at one of PPMD’s Race to End Duchenne events or a grassroots fundraiser, we are always looking for opportunities to bring families together for face-to-face interaction.
Through PPMD’s website, our social media presence, YouTube channel, and community site, we are able to share information, answer questions, and help connect you to other families who are on a journey similar to yours. As new social media platforms emerge, PPMD strives to find families wherever they turn for the latest information.
Community. Nothing is more important than knowing you are not alone. Let PPMD help connect you.