ONE VOICE Advocacy Summit: February 2011

Washington, DC.

The news from Washington, DC is constant with multiple agendas and many moving parts. Following the November elections of 2010, we have many new faces in both the House and Senate. The ripple effect of the elections is that committees will change, priorities will change, and many, many may not be familiar with Duchenne muscular dystrophy.

This year marks 10 years of advocating for Duchenne muscular dystrophy and while we have been very successful, our work is far from complete. Duchenne has changed over the last 10 years. Our sons are walking longer and living longer. Our base of knowledge about Dystrophin, protein-protein interactions, cellular pathways, and genetic modifiers is expanding. Biotech and pharmaceutical companies are engaged. We must continue the momentum.

In an effort to educate and engage new Members of Congress and demonstrate the impact of investments to date, PPMD will host a ONE VOICE Advocacy Summit in February. Utilizing the ACTION PLAN FOR THE MUSCULAR DYSTROPHIES, developed by the Muscular Dystrophy Coordinating Committee, this Summit will utilize the model used for Congressional Hearings. We will appoint a panel of experts to serve as facilitators. Panels consisting of government agencies (NIH, CDC, Department of Defense), academia, industry, patients, and families will be scheduled to discuss the plan, progress to date, priorities, and recommendations. It is our intention to review the plan, discuss investments to date in basic science, translation, care considerations, data collection, and newborn screening. As a community, we will outline priorities including gaps in care, the need for consensus around cardiac issues, and clinical trials. A full report will be published in Roll Call, a publication read by all Members and their staff.

In ONE VOICE, we will send a message (and published in Roll Call) to our champions thanking them for progress to date, asking them to continue this momentum, to help us accelerate the development of therapies, to one day soon see drugs approved as treatments for Duchenne. By reading Roll Call, new members will learn about Duchenne, about the amazing progress to date, how they can help maintain the momentum to accelerate therapies for Duchenne, and utilize this model as a road map for rare disease.

This promises to be one of our most exciting and ambitious Advocacy Conferences of the last ten years. We hope you will join us - whether a seasoned advocate or someone new to our advocacy program. Together our voice, our ONE VOICE, will be heard in Washington. Please add your voice to ours.


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