As I prepare for Thanksgiving, I run through a quick inventory in my head. I’ve got turkey and stuffing, a pumpkin pie with lots of whipped cream, and of course, Diet Coke. Most importantly, family and friends will be filling my home with laughter and love. Although I often wonder what would be different if Chris and Patrick were still here, I continue to find joy in all of my memories of Thanksgiving holidays as I make my own family’s favorite dishes.
I have to think my sons would be proud of what PPMD has become, and of the community created through our unwavering commitment to ending Duchenne. I know that I am. My gratitude extends to each one of you for the role you play in this fight we battle together.
Over the past year, we invested over $3.5 million in Duchenne research and related programs. We continued to focus on our Gene Therapy and Biomarker Initiatives. This year we began to learn more about barriers to inclusion for the underserved in our community. We submitted a nomination package to add Duchenne to the Recommended Uniform Screening Panel (RUSP) for newborns, and we continued to enhance the Duchenne Registry with the integration of Electronic Health Records.
We have made a $2 million commitment to ACTION through our Cardiac Initiative. We are prioritizing cardiac care and research, ensuring our loved one’s hearts grow Stronger With Every Beat.
Expanding certified care to more communities is a huge focus for PPMD’s Care team. Eight new centers were certified this year, bringing the total number of Certified Duchenne Care Centers to 35, with several more currently in the certification process. Through this network, we continue to increase our focus on cardiac care, psychosocial issues associated with dystrophinopathy, and treatment of carrier females.
We have been advocating in Washington, DC for more than 20 years. We continued the tradition this year through virtual visits to Capitol Hill with over 260 advocates representing 39 states. We also submitted an updated community-led Guidance to the FDA to ensure it reflects the progress and scientific advancements made since the first Guidance in 2014, as well as Gene Therapy and Cardiac Research, not included in the previous iteration.
Lastly, we harnessed the strength of our community via our first ever summit series on mental health for caregivers, siblings and individuals living with Duchenne, appropriately titled the Power of Us Summit Series. We convened over 700 attendees for our first hybrid Annual Conference, and provided valuable support and resources to underrepresented communities through the End Duchenne Tour. The number of PPMD’s Connect groups, PPMD’s Adult Advisory Committee (PAAC) members and newly diagnosed families engaged with us also continues to expand.
This Thanksgiving I am so grateful. For the families I have known since the beginning and those just finding their way to PPMD, for the healthcare providers who care for their patients with compassion and dedication, for our industry partners who truly exemplify the definition of partnership, for our researchers developing the therapies of the future, and for our donors who generously give to help fund our mission.
You each play a role in the fight to end Duchenne. And you make PPMD Stronger With Every Beat.
Founding President & CEO
P.S. I’m grateful to have you in my life. Take a moment to share on Facebook whose heart is special to you this Thanksgiving.