February 28, 2019 / Advocacy

PPMD Leads Duchenne Community to Nation’s Capital for Annual Advocacy Conference

Parent Project Muscular Dystrophy (PPMD) heads to Washington, D.C., March 3–5, for their annual Advocacy Conference. Over 100 families are expected to join PPMD on Capitol Hill to help inform Members of Congress about the critical need to continue investing federal resources in Duchenne specific priorities and programs.

Since the organization’s inception 25 years ago, PPMD understood the importance of government support in the fight to end Duchenne. “When Parent Project Muscular Dystrophy started in 1994, we knew that one of our first priorities was to travel to Washington, D.C. to give a voice to the thousands of families affected by Duchenne. We had to educate our nation’s leaders to get the federal support needed to make real progress in research and care,” according to Pat Furlong, Founding President and CEO of PPMD. “With a new Congress and many urgent priorities being communicated on the Hill, the timing of this year’s Advocacy Conference is crucial. We cannot let Duchenne funding get swept under the rug, we need to make our voices heard. We need to ensure that Duchenne is a priority across all government agencies.”

On Sunday, March 3, advocates will receive extensive training and support so that they feel empowered and prepared as they attend meetings with their Members of Congress. New families are paired with seasoned advocates and meetings are scheduled throughout the day on Tuesday.

This year, PPMD will host a Gene Therapy Policy Forum on Monday, March 4 as part of the Advocacy Conference. Gene therapy is an emerging, hopeful, therapeutic strategy currently being explored in Duchenne research. In early 2017, PPMD launched a Gene Therapy Initiative, identifying and supporting a number of gene therapy approaches. As part of this Initiative, PPMD decided to bring together regulators, companies, researchers, and most importantly the patient community to start the process of sharing information on the current status of gene therapies in Duchenne, understanding areas of collaboration, building relationships, and setting expectations.

Says Furlong, “A great deal has happened in gene therapy technology in a relatively short amount of time. In 2016, we held the first Gene Therapy Forum to work with regulators to understand the landscape and map out a pathway forward. Our March meeting in D.C. seemed like the right time to revisit our gene therapy landscape to understand where we are now and what else we must achieve in order to realize the full potential of gene therapy in Duchenne. The forum will bring together the researchers and clinicians leading this exploration and discuss the current status of clinical trials and research. PPMD plans to use this meeting as a jumping off point for a more in-depth look at gene therapy, that we will share with the community later this spring.”

In addition to Hill visits on Tuesday, PPMD will host a Congressional Briefing entitled Realizing the Promise of Patient-Focused Drug Development at noon in the Capitol Visitor Center (SVC 202). The goal of this briefing is to highlight the tremendous momentum within the drug development pipeline that has directly resulted from Patient Focused Drug Development (PFDD) policies and federal agency activities. Duchenne community stakeholders, including patient community members involved in clinical trials, industry representatives, clinicians, and patients receiving access to the first approved Duchenne therapies, will provide examples as to how the innovations resulting from PFDD pathways have impacted drug development, regulatory reviews, and access to new drug therapies.

Though registration is required for PPMD’s Advocacy Conference, there is no fee to attend. To learn more, click here.

If you are unable to attend the Conference in person, you can advocate from home. Click here to sign up for PPMD’s Advocacy Alerts and ensure that your voice, your story is included in PPMD’s messaging to lawmakers.

Join Our Mailing List