Parent Project Muscular Dystrophy (PPMD) fights to end Duchenne. We accelerate research, raise our voices to impact policy, demand optimal care for every single family, and strive to ensure access to approved therapies.
PPMD is proud to have played a vital role in every single victory against Duchenne since 1994 and our compassion, strength, and innovation continue to lead this community.
Advances in Research
For 23 years, PPMD has invested in every single therapeutic possibility for Duchenne. We take a cutting-edge approach to accelerate finding treatments that will end Duchenne for every single person impacted by the disease. PPMD has invested more than $50 million into Duchenne research. Our contributions over the years have led to advancements inspiring an additional $500 million investment in research from the federal government.
- We take a comprehensive view of the entire landscape to identify opportunities to accelerate the development of therapies and to ensure nothing is missed.
- We invest in innovative research to fight the disease, no matter the mutation, age, or stage of progression.
- We develop clinical trial methodologies and tools that address current roadblocks in Duchenne drug development. This includes innovative clinical trial designs that test multiple drugs, multiple arms, and multiple companies at the same time, enabling participation from infants to adults, minimizing exposure to placebo, and allowing success or failure answers more quickly.
- PPMD’s leadership in the Duchenne community created an inflection point, resulting in over 40 pharmaceutical companies now in the Duchenne space—companies who have invested approximately $8 billion in the fight to end Duchenne.
- Since 2016, two therapies have been approved in Duchenne with additional therapies approaching the regulatory finish line. PPMD plays a significant role in this process, convening thought leaders and community members to share their expertise and experiences with regulators and decision makers.
- We are the largest Duchenne-specific organization that focuses on high potential research opportunities that will impact every single person with Duchenne. We do not ask for payment. Our return on investment is simple: to End Duchenne.
Successes with Advocacy
Our advocacy efforts are focused on advancing the research that will lead to treatments and, ultimately, an end to Duchenne. We provide advocates with the tools and information they need to advance legislation and regulatory efforts that will impact their lives and ensure Duchenne is a priority across all government agencies. Perhaps no other rare disease health organization has come so far and so fast in the realm of Congressional advocacy than PPMD.
- Since the MD-CARE Act in 2001, over $500 million has been invested in Duchenne by the federal government.
- Our Advocacy Conference is the only officially organized gathering on Capitol Hill for the Duchenne community. It grows each year, with more and more attendees meeting their representatives and staffers, giving a face and a voice to PPMD’s advocacy agenda.
- We provide the community with the opportunity to raise their voices year round through Action Alerts—specific actions to take that influence legislators.
- We convene families for regulatory events, including federal Advisory Committee meetings and summits, designed to inform regulators about the Duchenne patient experience.
- We monitor broad national legislation that could impact our community and provide the Duchenne community with education, opportunity, and guidance to ensure that Duchenne community priorities are heard.
- We drive paradigm-shifting federal legislation and have changed the Duchenne landscape through the passage of five federal bills and Duchenne-specific Appropriations Report Language for more than a decade.
Improvements in Care
PPMD strives to ensure that people living with Duchenne are living longer, stronger lives, by helping them access expert healthcare providers, a comprehensive team of sub-specialists, and cutting edge treatments. We’ve seen some of the greatest advancements in the fight to end Duchenne around standards of care that significantly improve quality of life. PPMD has been at the forefront of those advancements.
- Since our founding in 1994, the average lifespan of people with Duchenne has increased from late teens/early-20s to mid-20s /early-30s. This incredible leap is not because of any drug interventions or treatments. It is because PPMD has pushed to advance care.
- In partnership with the CDC, PPMD advocates for and participates in the development, and updates, of standards of care for Duchenne. These guidelines outline the accepted treatments and therapies known to reduce Duchenne symptoms and improve quality of life.
- We help families access optimal, standardized Duchenne care and services via the PPMD Certified Duchenne Care Center Program. Centers that qualify for PPMD’s certification must meet and maintain the highest standards—complying with CDC care guidelines and applying new, evidence-based knowledge and care as it emerges. We have certified more than 18 care centers across the country and have plans to increase that number and include international centers, making comprehensive Duchenne care and services even more accessible.
- PPMD connects the Duchenne community through The Duchenne Registry (previously known as DuchenneConnect)—the only registry connecting Duchenne and Becker patients and families with clinical trials, care, and research. Many of these trials offer hope through treatments that improve function, prognosis, and quality of life.
- In collaboration with clinicians, researchers, industry, and legislative bodies, PPMD identifies existing gaps in care and research, then works to convene meetings which include global experts, around developing methodology to address, and potentially fill, those gaps.
- Decode Duchenne provides free genetic testing and counseling to people with Duchenne or Becker muscular dystrophy who otherwise could not afford genetic testing. Decode Duchenne is administered by The Duchenne Registry, a program of Parent Project Muscular Dystrophy, and is supported by Sarepta Therapeutics and PTC Therapeutics.
- PPMD helps people with Duchenne and their families understand the value and importance of standardized optimal care. We advocate for improvements in care and access for all families regardless of geography or socioeconomic status.
Connecting the Community
A Duchenne diagnosis can leave patients and families feeling isolated, overwhelmed, and confused. To combat that, PPMD connects our community to news, resources, advancements in research, and—most importantly—each other.
Over the years, we have created a robust and supportive Duchenne community that helps patients, families, and others in the Duchenne world connect—both online and offline.
- Our Annual Conference – the largest, most comprehensive, annual international conference focused entirely on Duchenne – gathers nearly 500 families each year to learn the latest progress in the fight to end Duchenne. They also gather for support, strength, and camaraderie, and to hear from families on similar journeys.
- In an effort to reach every single family facing a Duchenne diagnosis in the U.S., PPMD has launched a multi-year community experience called PPMD’s End Duchenne Tour. These free events bring updates on research, advocacy, and care to cities across the country.
- Our Online Community Forum allows families all over the world to connect with each other to chat, organize, and share stories, news, and information.
- We reach families locally through PPMD Connect (previously known as FACES), a parent outreach group. These groups meet in regions around the country, offering caregivers an opportunity to get in touch with those affected by Duchenne in their region. Together, those caregivers spread awareness of Duchenne, and help raise funds for the research, advocacy, and care work of PPMD.
- Our Race to End Duchenne endurance program (previously known as Run For Our Sons) brings hundreds of individuals and families together to participate in marathons, half marathons, family runs, 5Ks, and more to raise awareness of Duchenne around the country. Since it started in 2005, the Race to End Duchenne endurance program has raised over $10 million.
- In less than a decade, we’ve established parent outreach groups (PPMD Connect, previously known as FACES) in over 20 regions around the United States to advocate, fundraise, and educate about Duchenne locally.
Join the Fight
We are only as strong as our community. Join the fight, and together, we will end Duchenne.