Legislation & Public Policy Advocacy

PPMD drives paradigm-shifting federal legislation and has changed the Duchenne landscape through the passage of four federal bills and Duchenne-specific Appropriations Report Language (or federal funding) for more than a decade.

Ongoing Legislative & Public Policy Initiatives

• Federal Funding for Duchenne Research & Programs
• Healthcare Reform
• Duchenne Newborn Screening
• The BENEFIT Act
• Access & Coverage
• Advancing Patient Focused Drug Development (PFDD)

How to Get Involved

Each year, PPMD ensures that the Duchenne community is at the forefront of the hearts and minds of Congressional leaders and champions by convening our annual Advocacy Conference in Washington, DC at the start of the ‘annual Appropriations season.’ These days in Washington each year help to ensure that – while Duchenne may be a rare disease – we are central to the considerations of policy makers. Our PPMD Advocates are mighty and our presence in Washington D.C. has been central to our community’s success. But our grassroots outreach of friends and family who reach out to Members of Congress from home throughout the year is also critical to ensuring that Members of Congress view policies under consideration through the lens of the Duchenne community.

Legislative Advocacy Victories

By galvanizing the Duchenne-patient community and bringing our unified voice to Washington, D.C., we have fostered relationships with elected officials who became unwavering Duchenne champions on Capitol Hill, passed banner legislation, and established collaborations with every federal agency that touches Duchenne. As therapies have moved through development, we’ve told our personal stories to regulators and transformed those stories into quantifiable data. We have catalyzed research funding, and built a regulatory infrastructure for rare disease products that has reshaped the therapeutic, care, and trial environment.