Over the past three decades, PPMD has driven Congress to enact legislation that has transformed federal public health, research, therapy development and access policies to bring us closer to ending Duchenne.

This advocacy work has resulted in marked increases in Duchenne and related muscular dystrophy research funding, thorough public health surveillance to better understand the size and characteristics of the patient population, development and dissemination of evidence-based care standards to inform diagnosis and care, and policies to ensure the voice of the Duchenne and Becker community informs regulatory decision-making.

Thirty years after PPMD’s founding, more than 120 advocates – people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends will once again gather in Washington, D.C., to reflect on this milestone and, most importantly, continue fighting for additional progress. In addition to visiting with more than 120 Congressional offices, advocates will be participating in a special PPMD-hosted town hall focused on Duchenne clinical trials to enhance the trial participation experience.

“Never in my wildest dreams would I have believed someone if they had told me 30 years ago that the Duchenne and Becker community – led by PPMD – would have achieved the gains in public health and clinical care, medical research, and therapy development that we have achieved over the past three decades. I am so grateful to the thousands of PPMD advocates who have made these gains possible, especially all who have traveled to our nation’s capital year after year to share their stories with Congress and drive this progress,” said Pat Furlong, PPMD Founding President and CEO.

“As a result of this passionate commitment, we are fortunate to have seven FDA-approved therapies for Duchenne, including the first-ever gene therapy, in addition to more than three dozen potential therapies in various stages of development. Our efforts have helped increase the average life expectancy by a decade by making sure more clinicians have access to evidence-based standards of care and are thus better able to diagnose and care for individuals living with Duchenne and Becker,” Furlong added.

During the organization’s 2024 Advocacy Conference, PPMD advocates will be working to build on these gains by urging lawmakers to continue supporting critical research and public health programs at the National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and the Defense Department’s medical research function. This year’s request will also include provisions to intensify the NIH’s focus on cardiac health concerns of patients with Duchenne, a topic of increasing importance as people are living longer lives.

The 2024 policy agenda will also include provisions to expand CDC activities to understand the impact gene therapies and other treatments have across the lifespan as well as a continued campaign to strengthen FDA’s initiatives to heed the perspective of the patient and caregiver, including by enacting the BENEFIT Act that will update important benefit/risk laws to ensure patient experience data is part of that paradigm.

The clinical trials town hall session will feature patient or caregiver participants from Duchenne trials and will also include a representative from the FDA. The goal for the session is to generate specific recommendations that can be implemented to improve the experience of participating in these trials and to maximize the likelihood of effective trial outcomes. 

“I look forward to celebrating the 30th anniversary of our founding and the many, many people living with Duchenne and Becker, families, and friends who have helped us get to this point while at the same time redoubling our commitment to advocacy for the next 30 years and beyond,” Furlong said.

TAKE ACTION

During March 4-5, over 120 PPMD advocates — people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends will be participating in meetings with Congressional offices, urging them to support vital efforts that will support continued progress for our community. Click here to take action and help reinforce our message to Congress from home.