Our Advocacy Strategy & Impact

PPMD’s advocacy efforts are focused on advancing care and treatments for Duchenne by leveraging federal resources, building partnerships, and creating regulatory procedures and infrastructure. We empower advocates with the tools and information to engage with Congressional representatives to foster Duchenne champions within Congress and to advance legislation and regulatory policies, ensuring that Duchenne priorities are reflected across all government agencies.

Perhaps no other rare disease health organization has come so far and so fast in the realm of Congressional advocacy than PPMD. Since our advocacy efforts began in 2000, our community has helped to leverage over $800 million in federal funding into muscular dystrophy research, with over $600 million specifically for Duchenne, and achieved seven FDA product approvals.

Our Duchenne Advocacy Priorities:

• Increasing federal investment in Duchenne research
• Accelerating therapy development & product review processes
• Ensuring that patient experience is formally integrated into the drug development life cycle, from protocol design through payer determinations
• Improving healthcare access & delivery
• Developing newborn screening system & infrastructure
• Ensuring access & coverage to approved products
• Ensuring that regulatory & public policies reflect the priorities of adults with Duchenne

Legislation & Public Policy Advocacy

PPMD drives innovative, paradigm-shifting federal legislation and has changed the Duchenne landscape through the passage of five federal bills and annual Duchenne-specific Appropriations Report Language for more than a decade. Our Advocacy Conference is the only officially organized gathering on Capitol Hill for the Duchenne community. We also provide the community with the opportunity to raise their voices year-round through Action Alerts—specific actions to take that influence legislators.

Regulatory Advocacy

PPMD works to ensure that patient experience is central in product reviews. We convene families and other Duchenne advocacy organizations for regulatory events, including federal Advisory Committee meetings and summits, designed to inform regulators about the Duchenne patient experience.

Access & Coverage Advocacy

With the first FDA approvals of Duchenne therapies, the Duchenne community has entered a new environment for access and coverage. PPMD has been leading the effort to educate and guide the community through these unchartered waters.