Parent Project Muscular Dystrophy Advocates Urge Continued Funding in Washington

PPMD Advocacy Conference

Anthony Castle attends the Parent Project Muscular Dystrophy Advocacy Conference in Washington, D.C.

Nearly 80 parents, grandparents and relatives of boys with the most common form of muscular dystrophy attended the Parent Project Muscular Dystrophy 13th annual Advocacy Conference in Washington, D.C. in late February. The goal was to urge members of Congress to support critical research and patient care initiatives.

One of the main targets for the conferences was continued funding for Duchenne in three national organizations: the National Institutes of Health (NIH), the Centers for Disease Control and Prevention (CDC) and the Department of Defense (DOD).

“The conference was very successful. We are hoping to garner as much congressional support as possible in the coming weeks,” said Ryan Fischer, PPMD’s director of Outreach and Advocacy. “Right now we are focused on funding with agencies. We want to be sure the NIH, CDC and DOD are funded appropriately to continue the momentum we created when we started advocating over a decade ago. Since then, we've added 10 years to the lives of those diagnosed with Duchenne.”

In addition, PPMD and attendees asked that Congress

  • Include report language to support and advance the NeuroNEXT clinical trials initiative and Therapeutics for Rare and Neglected Diseases program as well as language to coordinate and link multiple Duchenne patient registry projects.
  • Oppose the president's attempt to eliminate the muscular dystrophy program and to use preventing and wellness funding to support basic operating programs at the CDC.
  • Expedite the process of reviewing Duchenne Care Considerations, identifying and filling gaps, and disseminating updates as broadly as possible to clinicians and impacted families.

The advocates from 23 states also requested provisions to strengthen the Food and Drug Administration's (FDA) commitment to advancing therapies for rare diseases.

Sen. Roger Wicker, R-Miss., and Rep. Doris Matsui, D-Calif., led the Duchenne sign-on letter, and Sen. Debbie Stabenow, D-Mich., presented PPMD's 2012 Congressional Champion award on Feb. 28.

PPMD is also focusing on a piece of legislation that will affect the entire rare disease community: the Prescription Drug User Fee Act (PDUFA) reauthorization. According to PPMD, the PDUFA allows the FDA to collect fees from drug manufacturers to improve and expedite the approval process for drugs. Learn more about PDUFA here.