In 1984, the word “Duchenne” entered my world, a life-changing moment when doctors could only offer the advice to go home and love my sons because they believed nothing more could be done. Since that day, I’ve dedicated myself to pushing the boundaries of our knowledge of Duchenne and providing hope for families like mine, fighting for every future.
Together as a community, we’ve made significant progress. Over the past three decades, PPMD has committed to extensive research, unraveling the complexities of Duchenne. We’ve tirelessly pursued groundbreaking discoveries, clarifying the role of dystrophin, the need to restore it, and the therapeutic approaches, such as gene therapy, that can achieve that goal. As we approach our 30th anniversary in 2024, our vision has expanded as gene therapy has transformed from a once-distant hope, into a tangible reality today for those living with Duchenne.
This year, our unwavering determination brought us to an extraordinary milestone: the accelerated approval of the first gene therapy for Duchenne. It’s a moment to celebrate, but we’re not at the finish line. I ask you to join me in securing a future where gene therapy becomes a reality for all who need it. Your gift to PPMD’s Gene Therapy Initiative by December 31st will play a pivotal role in achieving our $500,000 goal to drive research aimed at broadening access to gene therapy treatments.
With your support, PPMD will fund research to overcome pre-existing antibodies inotherwise eligible individuals. We’ll deepen our understanding of the long-term effects on the heart. Our team will actively pursue funding opportunities to advance next-generation and future-generation gene therapies and work to accelerate the path to repeat dosing. And as always, we’ll stand by families, guiding them through the complexities of this treatment journey.
In 2017, PPMD launched our Gene Therapy Initiative and we’ve since invested over $7 million in gene therapy approaches, including the early work that led to the development of the first gene therapy treatment for Duchenne. Through collaboration with experts and the insights of Duchenne families, we’ve identified challenges and opportunities for advancement within the field.
While our foundation is strong, our vision stretches to the next milestone.
This work is deeply personal to me. Your unwavering support empowers us to do something today that I couldn’t do for my sons so many years ago. Thank you for joining me in this fight – the fight to end Duchenne and the fight for every future.
Founding President & CEO