When I think about Duchenne research, I am awestruck. It is hard to wrap my head around how far we’ve come since my boys were diagnosed, but I also know we still have quite a distance to go in order to deliver transformative therapies for all individuals living with Duchenne.

Every success brings us one step closer to that goal. I am inspired by the science, and PPMD has supported research for years to move it from bench to bedside. I remain hopeful for more drug approvals and greater access. And, I look forward to the day each individual has the opportunity to pursue the treatments they need. The progress we’ve made in 25 years is remarkable, but it is not enough.

Gene therapies and gene editing strategies are transformational possibilities. These possibilities, however, come with several obstacles on the pathway to making them available to every person living with Duchenne. We will approach these challenges from every angle, the way we always have.

Together, with your support of PPMD’s Gene Therapy Initiative, we will work toward finding solutions that allow for all of those diagnosed to safely receive these therapies. Donate today and help us turn obstacles into possibilities.

In early 2017 PPMD launched our Gene Therapy Initiative, a long-term concept that seeks to accelerate the potential of Gene Therapy as a therapeutic for Duchenne. Our early strategy was to bring attention to and fund key questions that must be answered in order for the technology to progress toward approvals. Through years of hard work and determination, we have reached that point. We now have multiple companies with clinical trials delivering Gene Therapy to a significant number of boys, with additional companies bringing forward new Gene Therapies on the horizon.

However, our biggest question remains: How do we make Gene Therapy accessible for all who need it?

PPMD has invested in new research and additional work to address that critical question, increased focus on the quality of care for those with Duchenne, and targeted advocacy efforts to put the patient voice at the forefront of drug-development and regulation. We also plan to convene a consortium of expert researchers and clinicians to attack these challenges and help our community move forward with next steps and additional solutions.

We need to understand the sustainability of these treatments for those who have been dosed. Will redosing be needed? And how will we know the timing for redosing? We need to add additional trials to our pipeline to ensure more inclusivity for non-ambulatory, older patients and those with Becker muscular dystrophy. And, perhaps the most difficult question of all, is how to deliver Gene Therapy when the individual has neutralizing antibodies to the viral delivery method. What are the best approaches to decrease circulating antibodies and enable those individuals to access gene therapies?

We will bring together the best and the brightest in their fields to help us find these answers and move forward. Support PPMD’s Gene Therapy Initiative today and your gift will drive forward progress in overcoming these obstacles.

Thank you for your support and your trust. We will face the challenge together and find our path toward possibilities for all.

Sincerely,

Pat Furlong
Founding President & CEO

P.S. Supporting PPMD’s Gene Therapy Initiative this spring will help drive us forward on the path to ensuring Gene Therapy is an option for everyone. Join us with a donation today.

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