Today is one of my favorite days of the year. Thanksgiving is a holiday that revolves solely around friends and family coming together to just eat, maybe have a little wine, and then eat some more. And, it’s a holiday that is built around one theme — gratitude. I hope wherever you are, whoever you’re with, whatever you’re doing, you know how extraordinarily grateful I am for you, your family, and your ongoing support of PPMD and our fight to end Duchenne.
For 25 years, this community has been at the top of my list of things for which I am thankful. The support, passion, commitment, and love of this community transcends anything we could have imagined when a group of parents and grandparents first came together in 1994.
This has been another year of ups and downs for our community – hope and frustration, joy and sadness. And yet it is astounding to me, despite all of the raw emotion that comes with each and every day, how much this community can accomplish in a year. We continue to learn from each roadblock, discovering new approaches and addressing the breadth of this complicated disease.
As I sit down to dinner with my family today, I am so proud of where we are now versus where we were when PPMD first started and I am in awe of the accomplishments of this community and our continued momentum:
- PPMD’s investment of over $3.7 million in research grants and projects in 2019 alone.
- A $5.1 million investment in gene therapy and a $1.8 million investment in cardiac-related therapies, as well as a commitment to supporting promising early-stage preclinical work so that innovative and bold approaches to treating Duchenne receive critical funding.
- The certification of five more care centers (including our first adult center) bringing our prestigious list of clinics to 28.
- An updated Duchenne Registry with an app-based technology that empowers patients and families to be citizen scientists from the palm of their hands, sharing your experiences with care providers and companies to better understand the disease and inform the architecture of future trials.
- The exploration of a platform trial design to minimize the use of placebos in the clinical trial process.
- Launching a newborn screening program that will help identify babies affected by Duchenne in a state with a high birth rate, working toward national adaptation, and eventually the possibility of early interventions.
- Five years of free genetic testing and counseling through our Decode Duchenne program.
- A new interactive Drug Development Pipeline tool that enables families to stay up-to-date on all therapies that are currently in development — a pipeline full of promise because of the investment PPMD has made in research the last 25 years with your support.
Perhaps most importantly, I am so proud of how PPMD is able to bring the community together. This year, we connected more families than ever before with expert physicians, researchers, and care providers across the country through live and virtual educational events, including our Annual Conference — the largest Duchenne-specific conference in the world and our End Duchenne Tour.
PPMD and the families that connect with us are my extended family. I so appreciate every family that hosted a fundraiser, joined us in tackling Duchenne for the 12th Annual Coach To Cure MD, and participated in one of our ever-expanding Race to End Duchenne events.
We are where we are today because of people like you — families that continue to go above and beyond to join the fight, to fund the fight, to help us end Duchenne. Families that believe today is better because we have each other. And it’s because of you that we are confident that tomorrow will hold more promise than ever before. The future for everyone living with Duchenne has never been stronger. For that, I am truly grateful.
Happy Thanksgiving from my family to yours!
Founding President & CEO
P.S. Take a moment to share on Facebook what your heart is grateful for this holiday season — a time of joy, a time of love, and a time with family.