As I prepare for another holiday to begin, I am again overwhelmed with gratitude for the compassionate, resilient community we’ve built through PPMD. You are each an extension of my family, the family we have created together. You bring light and hope for the future as we continue to fight.

Over the past three decades, we have accomplished so much together and I know that every win—no matter how small or large—brings us closer to the day where we end Duchenne. We now stand at the threshold of a new era, poised to move science further, faster; for research to evolve. Our progress is a testament to you, and I am incredibly grateful for your unwavering commitment to our cause and the pillars that guide our mission.

Research

2024 was a monumental year not only for PPMD as we celebrated our 30th anniversary, but for the entire Duchenne community. We reached significant milestones when the FDA expanded the label for ELEVIDYS™ and when we gained the eighth therapy approval for Duchenne with DUVYZAT™. Over the past year, PPMD has invested more than $4 million in research funding across the development pipeline – from basic research to venture investments to help accelerate development.

Our commitment to support research across all stages extends beyond research grant funding. Through The Duchenne Registry, the Duchenne Outcomes Research Interchange, and PPMD’s Electronic Health Record study, we are collecting valuable data to help us all better understand Duchenne.

Care

PPMD continues to convene the brightest minds in care.The year began with the annual Duchenne Healthcare Professionals Summit and included a collaborative workshop on Becker muscular dystrophy and consensus meetings on swallowing, gastrointestinal and genitourinary care. Throughout the year, members of the PPMD team continued to work alongside the OPTIMIZE-DMD consortium, a group of endocrinology experts working to establish updated standards of care for bone health and other areas of endocrinology care. As part of our ongoing Cardiac Initiative, PPMD held the third in a series of cardiac workshops, this year focusing on rapidly changing cardiac standards of care and post-gene therapy monitoring, and brought together leading experts to discuss access to heart transplantation.

Advocacy

PPMD’s impact in Washington, DC, has continued to grow over 20 years since launching our federal advocacy efforts. This year, we also invested in an inaugural state advocacy program beginning with a pilot of eight states. We are empowering advocates to use their voices to educate local policy makers and affect change at the state level.

As the sponsor of the Newborn Screening Package for Duchenne to the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), PPMD continues to lead the effort to add newborn screening to the Recommended Uniform Screening Panel. While we anticipate a vote in the spring of 2025, we have already shifted focus to state activation and implementation so that when newborns with Duchenne are identified, there are tools and resources for everyone – clinicians, parents, and infants diagnosed.

Community

We joined forces in meetings, workshops, races, fundraising events, and Connect gatherings, with the number of individuals engaged in our community growing every day. Our Annual Conference in Orlando saw a record-breaking year of attendance with over 1,400 attendees, including over 100 teens and adults living with Duchenne and Becker. We also marked our 30th anniversary by launching PPMD Together, our re-imagined regional family workshop series that has been met with widespread enthusiasm and support from our community.

Together, we are fighting for every future. Thirty years is our new beginning.

This video serves as a brief, yet poignant reflection on the progress we’ve made as a community over the past 30 years, as well as a call to action and source of inspiration as we embark on this next chapter, together.

 

This Thanksgiving, my heart fills with gratitude for the families who have been with us since the start and those who are just finding their way to PPMD, the dedicated and compassionate healthcare providers, our industry partners and researchers developing the treatments of the future, and our generous donors who support our mission.

Happy Thanksgiving,
Pat Furlong
Founding President & CEO