Parent Project Muscular Dystrophy is leading the fight to end Duchenne.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.

• In the past 18 months, we’ve funded 26 research projects totaling $3.4 million.

• 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.

• 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)

Research Advocacy Care

2011-2012SNAPSHOT

• Over $250 million leveraged in NIH, CDC, and DOD funding for Duchenne.

• We annually push to expand funding at the NIH, CDC, and DOD for Duchenne.

• 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.

• 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.

Parent Project Muscular Dystrophy is leading the fight to end Duchenne.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.

• Through our website, community site, Facebook, Twitter, and mobile app, we continually work to educate you about our most pressing needs: care and research.

• Our monthly webinar series provides direct access to top researchers, clinicians, and industry. Links to these webinars are provided immediately afterwards, and your questions are passed along to the presenters.

• New care materials: » fact sheets about endocrine

(hormone) issues for people who take corticosteroids

» body system fact sheets » updated and more user-friendly care

website

• New research fact sheets about most of the drugs or devices that are in clinical trial or are close to being in clinical trial.

• Finishing up the Neuromuscular Task Force project: a cooperative agreement with the CDC to develop materials that will let pediatricians, family doctors, and therapists identify muscle weakness earlier.

• We continue to offer materials related to Duchenne, care, and research, free of charge to families and institutions that request it.

• Our educational series, the Connect Conference and Meetings, is an opportunity for families, physicians, researchers, and experts of all kind, to speak face-to-face about Duchenne. These meetings open communication channels, lay the groundwork for future collaborations, and shape our understanding of the Duchenne landscape.

Education Fundraising Community

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$• In 2011, we launched the NEXT Major Gift

fundraising campaign to help support high impact Duchenne research grants. This three-year, $10 million campaign continues to push forward having provided nearly $6 million to numerous research initiatives this far.

• Run For Our Sons (RFOS) is a national endurance program developed to raise awareness and funds for Duchenne. RFOS is an official charity in some of the most prestigious races in the country, including the ING New York City Marathon, Bank of America Chicago Marathon, and both the Walt Disney World Marathon Weekend and the Disneyland Marathon. RFOS raises over $1 million annually.

• In addition to marathons, RFOS expanded its endurance portfolio in 2012 to include the NYC Triathalon and Conquer the Canyon, a 3-mile hike into the Grand Canyon.

• Since 2008, we have partnered with the American Football Coaches Association each September on Coach To Cure MD, a national awareness campaign that has raised $1 million to date. With over 500 colleges, universities, and high schools participating, Coach To Cure MD illustrates the unique parallels between Duchenne and college football, a game where young men are at the peak of their muscle strength.

• Our Grassroots Fundraising Events continue to be the heart and soul of community with over 120 events in the last two years.

• We continue to offer a wide variety of fundraising activities, including: » ShopForOurSons.org (a partnership

with Amazon) » United Way » A robust matching gift and employee

gift program » STIR (PPMD's monthly giving campaign)

• FACES, the official parent-led outreach initiative of PPMD, provides family mentoring, supports national awareness and advocacy campaigns, and helps raise money for PPMD. There are currently 11 FACES groups around the country, including, most recently Northern California and Ohio.

• FACES groups continue to grow, hosting monthly meetings and events across the country.

• Each year nearly 500 families from around the world gather at our Annual Connect Conference to learn the latest progress in the fight to end Duchenne. They also gather for support, strength, and camaraderie. This is your opportunity to gain direct access to the people fighting everyday for children like yours and meet and reunite with families on a similar journey as you.

• This year, we expanded our Connect series, to include a West Coast Meeting in San Diego.

• We launched HerSelf First, a program that helps women caregivers invest in their well-being on a daily basis. There is a direct relationship between a caregiver’s well-being and her loved ones’ quality of life. When a caregiver can put herself first, everybody benefits.

• Our community site (over 3,600 members and 1,900 discussions) allows families all over the world to connect with each other to share stories, news, and information.

• We continue to connect with you through your favorite social media channels: » Facebook.com/ParentProjectMD

(over 11,600 likes) » Twitter.com/ParentProjectMD

(over 1,000 followers) » Youtube.com/ParentProjectMD

(53 videos and over 405,400 video views)

Parent Project Muscular Dystrophy is leading the fight to end Duchenne.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.

2011-2012SNAPSHOT