Turning obstacles into possibilities is what initially lead me to joining PPMD. I had little personal experience with our Duchenne community before I began working with Dr. Jerry Mendell and a number of other fantastic providers. While I was drawn to Duchenne research because of the exciting work being done in the field of Gene Therapy, after meeting the individuals in this community my interest and focus shifted to supporting these scientific breakthroughs to ensure every individual with Duchenne has the opportunity to receive innovative and effective therapies.

More than anything, if the promise of this research is realized following clinical trials, I want to see Gene Therapy become an accessible option for all who need it. I can see the potential ahead, but first we must overcome the obstacles in front of us. Make your gift today to support our efforts.

Duchenne research has progressed significantly over the past several years, but we need to keep the momentum moving forward. We need to develop impactful therapies and ensure that everyone living with Duchenne is provided the opportunity to receive treatments.

PPMD is currently funding two projects focused on immune response with Dr. Kanneboyina Nagaraju. One study is nearing completion and will help to inform us if there is any potential immune response to newly produced dystrophin, as well as drugs that could help prevent such an immune response. The second study, recently announced in collaboration with Duchenne UK, looks to address the initial immune response the body has to AAV, investigating ways to mitigate that initial immune response and enable more AAV to reach the muscle cells. We have also provided bridge funding to Dr. Dongsheng Duan to continue his work on understanding the immune response to future strategies aimed at systemic delivery of CRISPR/Cas-9.

PPMD also participates in the Working Group on Pediatric Gene Therapy and Medical Ethics (PGTME) at New York University, through funding and work group participation. The mission of the group is to advance research policy and education regarding ethical issues surrounding gene therapy trials. The working group seeks to promote improved understanding of challenges and initial best practices for ethical research practices across the evolving landscape of genetic technologies.

These projects will hopefully bring us some of the answers we need, but we recognize more answers will be needed to guarantee all those living with Duchenne can have access to safe and effective therapies. Just as PPMD has done for more than 25 years, we will bring together the best and brightest researchers and clinicians to collaboratively overcome any obstacles in our way. Your gift today will lead to tomorrow’s possibilities.

Upcoming Webinar

Join us for a Zoom panel discussion on Tuesday, April 20 at 1 PM EDT to discuss the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments.

Moderators:

• Pat Furlong, Founding President and CEO, PPMD
• Eric Camino, Vice President of Research and Clinical Innovation, PPMD

Presenters:

• Dr. Kanneboyina Nagaraju, Founding Chair, Pharmaceutical Sciences, Binghamton University
• Dr. Perry Shieh, Department of Neurology, Ronald Reagan UCLA Medical Center

Give your gift today to support PPMD’s Gene Therapy Initiative. Together, we will turn obstacles into possibilities.

Thank you for supporting innovative research and for trusting PPMD. We are on the path to possibility.

Sincerely,

Eric Camino, PhD
Vice President of Research and Clinical Innovation

P.S. Your investment in PPMD’s Gene Therapy Initiative brings us closer to turning obstacles into possibilities. Join us with a donation today.

Donate >