November 24, 2021 / Community

This Thanksgiving I am grateful for you

#PPMDPowerOfUs Collage

The turkey is prepped, and we are ready to turn on the oven bright and early tomorrow. My emotions are always heightened  during the holidays, but this year it’s even more palpable. The holidays are bittersweet, there are always two chairs missing from the table and I think about the what if’s, the should have beens…

But after this past year, the joy of togetherness far outweighs the grief. Fear and isolation are giving way to hope, promise and gratitude.  As Duchenne families, we understand this dichotomy all too well. Pain and prosperity, success and sadness, looking toward the future and living in the present. 

We also understand the value of our community, our support system, our people. You are my people and today, I am especially  grateful for each one of you!

This year we have accomplished incredible things, and I am proud of the power we have when we work together. We have committed over $3.5 million to Duchenne research, continuing to focus on our three key initiatives: Cardiac, Gene Therapy and Biomarkers, as well as areas that support our research such as the Duchenne Registry and Newborn Screening.  We launched our EHR Integration Pilot and plan to have data flowing into the Duchenne Outcomes Research Interchange before the end of the year. The Newborn Screening Pilot recently completed two years of ground-breaking recruitment with more than 36,000 babies screened for Duchenne in New York State. As a result of screening, four babies with Duchenne or Becker and one carrier female were diagnosed and provided with optimal early care. 

With the pandemic still a reality, our Care team has continued to provide expert guidance for the community while navigating the COVID-19 vaccine roll-out and often changing medical guidelines. Through our network of Certified Duchenne Care Centers we have increased our focus on cardiac care, psychosocial issues associated with dystrophinopathy, and treatment of carrier females.  

I was impressed to see the largest number of advocates ever join us for this year’s virtual Advocacy Conference with 435 individuals representing 43 states. Advocates participated in over 300 virtual meetings with their local representatives. This year we are also investing resources to bring together members of our community, including experts in their fields and patient families, to update the community-led Guidance, originally submitted to the FDA in 2014. New this year is the addition of a Pharmaceutical Advisory Board and a Cardiac and Gene Therapy Working Group. The work we are doing in Washington, D.C., will have a significant impact on the future of drug development for Duchenne and lead the way for other rare diseases.

Even from a distance, our connection to one another is stronger than ever. With virtual programming we were able to overcome barriers to participation faced by many in our greater community. In June, we had over 1,700 attendees, representing 51 countries at our Annual Conference. Our Sibs Connect group, Newly Diagnosed and teen/adult  groups have benefited significantly from the virtual participation format as well. And just a few months ago we launched our Podcast series which is another space for us to talk about our journey. 

Each step we take brings us closer to the day we will end Duchenne and closer to each other as a community. When my boys were first diagnosed, life felt hopeless. But we found one another and we built PPMD together. It was the power of each of us that made today a possibility for our Duchenne community. 

This Thanksgiving, tell us who you are grateful for and why. 

Spread your thanks by sharing a photo or image and thanking someone… someone in our community, someone in your village, or someone that has made a small gesture that has helped you get through a hard day. Share your photos and images on social media using the hashtag #PPMDPowerofUs.  Every image posted with the tag #PPMDPowerofUs will populate an online gallery here, so that we can all revel in the power of each other this holiday season. 

Thank you for being my people. 

Happy Thanksgiving, 

Pat Furlong
Founding President and CEO 

P.S. Share who you are grateful for with the hashtag #PPMDPowerofUs I can’t wait to see the power of us come together. 

Join Our Mailing List