by: Parent Project Muscular Dystrophy
For over three decades, my life’s work has centered around scientific research aimed at making a positive impact on the lives of individuals living with Duchenne. I have witnessed breakthroughs as therapeutic options moved from research labs into clinics, and I have had the opportunity to contribute to the growth of this field by exploring many of these, including gene therapy.
As the Chair of PPMD’s Scientific Advisory Committee, I’ve been immersed in the constantly evolving possibilities for the Duchenne community. I’m proud to have seen first-hand how your belief in PPMD made our first systemic gene therapy program a reality, and I am excited for what the future may look like from here.
My collaboration with Pat Furlong started in the early days of PPMD. Pat shared a powerful vision – a vision of fighting for every future – which resonated with me deeply. A day where therapeutic options would be accessible to every person living with Duchenne.
Your gift by 12/31 to support PPMD’s Gene Therapy Initiative is a commitment to research, increasing access, and reaching the next milestone. Give today.
The progress we’ve made in gene therapy would not be where it is today without the generous support of individuals and families in the Duchenne community, along with the visionary leadership of PPMD. That unwavering passion to fight for every future has resulted in PPMD’s investment of over $7 million in gene therapy research spanning nearly 30 years since its founding.
The organization takes a cutting-edge approach to find treatments that can truly make a difference for every single person impacted by the disease. PPMD’s Gene Therapy Initiative is a reflection of this passion for innovation and determination to address key questions that must be answered in order to advance the technology needed to benefit all individuals living with Duchenne.
While we have reached a remarkable milestone, there are still significant challenges ahead in the field of gene therapy. Give today and your support will be an integral part of work that will impact this generation of patients.
With your support, PPMD will fund research to overcome the numerous challenges that we still face in this burgeoning field. PPMD’s Gene Therapy Initiative aims to help us all deepen our understanding of the long-term effects of this treatment on the heart, prioritize research for next-generation and future-generation gene therapies, continue our work navigating the challenge of pre-existing antibodies so they are not a barrier to otherwise eligible individuals and accelerate the path to repeat dosing.
Our work is far from over. Together, we have an opportunity to drive research aimed at broadening access to gene therapy treatments. Let’s achieve the next milestone in gene therapy, advancing technology towards muscle function preservation and quality of life for generations of individuals living with Duchenne to come.
Warmly,
Lee Sweeney, PhD
PPMD’s Scientific Advisory Committee Chair
