Earlier this month, more than 120 PPMD advocates – people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends – from 26 different states gathered in Washington, D.C. to urge legislators in the U.S. Senate and House of Representatives to keep Duchenne and Becker at the forefront of their minds when making policy decisions. 

The first day of activities during PPMD’s 2024 Advocacy Conference focused on equipping PPMD advocates with comprehensive knowledge and resources necessary for their meetings on Capitol Hill. This year’s request to Congress included:

• Sustained investment for vital research and public health initiatives at the National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), and the Defense Department’s medical research arm. 
• Emphasis on the importance of directing NIH attention towards cardiac health issues in Duchenne patients, an increasingly significant concern given the extended life expectancy of affected individuals.
• Advocating for CDC efforts to comprehend the lifespan impact of gene therapies and other treatments.
• A continued push to empower the FDA to prioritize patient and caregiver perspectives. This included advocating in support of the BENEFIT Act to update benefit/risk laws, ensuring patient experience data plays a central role in regulatory decisions.

Before heading to Capitol Hill for meetings with congressional offices, advocates came together for a pivotal Town Hall session, titled “Clinical Trials – Past, Present & Future – Checking in on Meaningful Outcomes for Families.” This session featured insightful discussions from parent and patient panels, as well as representatives from the FDA’s Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), and a neurologist from one of PPMD’s Certified Duchenne Care Centers, the University of Rochester Medical Center. Participants emphasized the need to consider stability as a benefit when evaluating potential treatments. These important conversations are paving the way for increased engagement between our community and the FDA and other stakeholders, ensuring that trial outcomes align closely with the needs of those affected and are informing PPMD’s patient preference work for the year. 

Over the course of three days , PPMD advocates tirelessly raised their voices on Capitol Hill to champion our priorities and uphold our community’s interests. Those unable to attend in person rallied by sending over 1,500 letters to Congress, amplifying our collective voice. If you haven’t had the chance yet, there’s still time to take action and make your voice heard by submitting a letter to your members of Congress here.

United in our fight for every future, our voices continue to resonate throughout Capitol Hill, amplifying awareness and driving meaningful change for the Duchenne and Becker community. Thank you to all the advocates who participated in this year’s Advocacy Conference, as well as everyone who has taken action from home! We look forward to continuing to advance our community’s goals with Congress and federal agencies, together.