Parent Project Muscular Dystrophy is leading the fight to end Duchenne.
2011-2012 SNAPSHOT
We see a future without Duchenne, and PPMD, along with this amazing community is doing everything it can to make it happen. We believe it takes a comprehensive approach to best serve the Duchenne community. Everything we do and everything we ask you to do, is to serve one goal: End Duchenne.
Research
Advocacy
• In the past 18 months, we’ve funded 26 research projects totaling $3.4 million.
• Over $250 million leveraged in NIH, CDC, and DOD funding for Duchenne.
• Our direct research investments have helped to leverage additional private/ public support, to include NIH grant awards of $30 million in the past 3 years.
• We annually push to expand funding at the NIH, CDC, and DOD for Duchenne.
• Currently funding 21 active research projects at $4.5 million which cover a broad array of Duchenne research strategies and approaches. • Directly supporting four clinical trials and providing travel funds for two more. • We support the TACT review process to better assess Duchenne research proposals and help to expedite these projects to clinical trials. • Our Scientific Advisory Committee is comprised of 10 of the leading Duchenne authorities from around the world. Since 2010, they have reviewed 54 grants. • Launched Review of Approved Drugs for Duchenne (RADD) Working Group, a PPMD-funded three-way collaboration between Nationwide Children’s in Columbus; Children’s National Medical Center in Washington, D.C.; and TREAT-NMD to prioritize FDA-approved drugs for further testing. • Partnered with The Public Library of Science (PLoS) to launch a new publishing initiative called PLoS Currents: Muscular Dystrophy, in order to accelerate the pace of muscular dystrophy research through the rapid sharing of new findings and ideas. • PPMD President Pat Furlong serves on: »» National Organization for Rare Disorders (NORD) Board of Directors »» Institute of Medicine’s (IOM’s) Committee on Pediatric Studies »» Chairs Congressionally Directed Medical Research Program in Duchenne for the Department of Defense (DOD)
• Board approved FDA Policy to identify the needs and barriers for developing clinical trials. • Held a high impact, landmark meeting with FDA leadership to discuss FDA policy and the unique challenges with clinical trials for Duchenne. Ongoing discussions are planned. • Advocating for rare disease provisions in the Prescription Drug User Fee Act. Specifically developing Fast Track and Accelerated Review guidance for small patient populations and deepening the patient engagement in the review of medical products. • We continue to voice concern and play a leadership role in trying to stop the possible consolidation of critical Duchenne related programs at the Center for Disease Control. • Hosted One Voice Summit in 2011 to analyze progress on care and research since the MD-CARE Act passage in 2001. • Published a report with findings from the One Voice Summit to inform the next update to the MD Action Plan developed by the Muscular Dystrophy Coordinating Committee. • As the first muscular dystrophy-specific legislation ever to pass through Congress, the MD-CARE Act remains a cornerstone of Duchenne advocacy in Washington. PPMD has led the efforts to pass (2001) and then reauthorize (2008) the bill to ensure Duchenne remain a priority to the federal government. We are laying out the strategy for the 2013 reauthorization.
Care • We have brought together clinical and family experts to make recommendations about, and identify areas of need related to Cardiac Care, Behavior, Adjusting to the Diagnosis, Endocrine, and Metabolism since 2006. • Host of two-day workshop on Transforming Duchenne Care, bringing together leadership from top medical institutions, neuromuscular experts and thought leaders, and patient representatives to discuss in an open forum the ideal configuration, features, services, and structure of state-of-the-art family centered care for patients with Duchenne. • A partner in advocating for and developing the Duchenne Care Considerations, which are distributed without charge to families, institutions, and the medical community. • Engaged with CDC and the Duchenne and Becker community to plan for updates, fill in gaps, and evaluate the impact on care and health outcomes. • Conducting a survey to understand the needs and strengths of mothers of individuals with Duchenne and Becker, to help guide PPMD’s programs for mothers. • Our registry, DuchenneConnect, gathers information from individuals with Duchenne and Becker, and from their parents, to learn more about health and care use. We gather information useful to researchers, industry, clinicians, and families.