Thanksgiving offers each of us a moment to reflect on all that we are grateful for. This year, I am filled with gratitude for the impact we have made on the Duchenne landscape these past three decades, and for the way this community has enriched my life. You have each become members of our cherished family.

And this year, our family has much to celebrate. Although the holiday season without Chris and Patrick is always bittersweet, I believe my boys would be profoundly proud of how far we’ve come in the last 30 years. Our community has never experienced so much momentum, and it’s all thanks to your unwavering support.

Research and Breakthroughs

Our determination to fight for every future drives our commitment to funding research. Over the past year, PPMD has invested over $4 million in research funding, largely to support our Gene Therapy and Cardiac Initiatives. Our collective community celebrated two additional drug approvals, including the first accelerated approval of a gene therapy for Duchenne. We are proud to have been supporters of the early work that led to both of these therapies.

Data and Diagnosis

Our commitment goes beyond research funding. It extends to connecting researchers with data to help speed the development of new therapies, and advancing work to ensure early, equitable diagnosis. We’ve witnessed significant progress for Duchenne newborn screening, with a recommendation for evidence review at the federal level and approvals at the state level in Ohio and New York, as we continue advocating for similar legislation in more states.

This past year also marked a milestone for The Duchenne Registry, highlighted with the release of the Fifteen Year Registry Report, summarizing the data of approximately 5,500 registrants from nearly 125 countries. I can’t say thank you enough to each individual and family who has participated in The Duchenne Registry. The contributions you’ve made as citizen scientists have been instrumental in advancing research and understanding of the disorder.

Care and Collaboration

Our dedication to address Duchenne from every angle continues to drive forward meaningful initiatives. This year, we continued our work with leading cardiologists in the ACTION network, building upon our commitment at the end of 2022 by convening the second part of a three-part cardiac workshop series. We also brought together global leaders in Endocrine care twice to address key areas such as steroid use, bone growth, and the critical need for data.

Community and Unity

Throughout the year, we’ve united as a community, both in virtual spaces and in countless cities throughout the country. We joined forces in meetings, workshops, races, fundraising events and Connect gatherings. Our advocacy work, spanning over two decades in Washington, D.C., returned in-person to Capitol Hill for the first time since the pandemic. And, PPMD’s Annual Conference in Dallas saw record-breaking attendance with nearly 1,100 participants, including 133 teens and adults living with Duchenne or Becker.

From every corner of Duchenne, we are fighting for every future.

This Thanksgiving, I am immensely thankful for our wonderful community. I cherish the families who have been with us from the start and those just finding their way to PPMD, the healthcare providers who care for their patients with compassion and dedication, our industry partners who exemplify the definition of partnership, the researchers changing what the future means for Duchenne, our champions in Congress advocating for our community, and our generous donors who power our mission.

Thank you for your continued support. Together, we will end Duchenne.

Happy Thanksgiving,
Pat Furlong
Founding President & CEO