PPMD Annual Report - 2012

Because we always knew what was possible.

We’re finally seeing the impossible happen.

Tim, age 14 and his younger brother John, age 3.

PPMD’s Progess Report

Dear friends, families, and supporters, More often than not, the difference between the impossible and the possible is sheer determination.

That’s especially true for our community.

We’ve accomplished so much together. When I think of all that our community has made possible over the years, all that we accomplished in 2011/2012 (outlined in this year’s Progress Report), and all that we’re doing today, my heart fills with appreciation and gratitude for each and every one of you—for your passion and commitment.

Through our hard work and dedication, as well as that of clinicians, researchers, and industry who have joined our efforts, we’re finally seeing some of the moments we never thought we would see. Life with Duchenne is much different than it was when my sons were diagnosed in 1984. Today, people with Duchenne are walking longer than ever before, they’re graduating from college, they’re getting married, and they’re starting careers and families of their own.

And we’re still going. In 2012 alone, we saw a number of unbelievable achievements. We expanded our partnership with the FDA, holding two important meetings in 2012 to help provide them with the information about Duchenne that they need—about both the disease and the community—to exercise flexibility in the review process. We saw not only a record number of clinical trials, but also trials that are showing signs of efficacy and moving forward in the drug approval process. And we built an unprecedented network of experts in research, drug development, regulatory compliance, and advocacy that are dedicated to helping us achieve our research goals.

Behind this amazing progress are the families and friends like you who roll up your sleeves and do anything and everything you can for people with Duchenne. There are so many ways that we can make a difference for the Duchenne community: from making the choice to enroll your child in a clinical trial or other clinical research to taking part in our advocacy efforts to completing surveys and connecting with others through our FACES groups.

The tools we need to defeat Duchenne continue to expand. Social media has proven to not just be a fun way to reconnect with old friends or share funny videos, but also a resource that helps us share critical information in a moment’s notice and connect with one another. And combined with the success of programs like Run For Our Sons, Coach To Cure MD, and numerous grassroots fundraising events, it is how we can educate the global community and raise awareness.

We will not rest until we end Duchenne. But with you on our team we know that we will continue to see the impossible become possible.

Warm regards,

Pat Furlong, PPMD President & CEO

Who we are Parent Project Muscular Dystrophy’s mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community.

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About us Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne.

Board Robert J McDonald, MD Board Chairman

Staff John Hiatt Howard Kaplan*

John Killian Board Treasurer

Martin Karlin

Daniel Garofalo Board Secretary

Robert Nutt

Heinrich Meermann

Doug Biggar, MD

Christine L. Piacentino*

Neil Brandom

James Poysky, PhD

Catherine Jones Collins

Donna Saccomanno Co-Founder*

Anessa Gaydou-Fehsenfeld Bob Getler Lance Hester

*lifetime, non-voting member

Jasbir Seehra, PhD Ellen Wagner

Pat Furlong Founding President, Chief Executive Officer Kimberly Galberaith Chief Operating Officer Sharon Hesterlee, Ph.D. Vice President of Research Holly Peay, MS, CGC Vice President, Outreach & Education Will Nolan Director of Communications & Administration Ryan Fischer Director of Community Outreach & Advocacy Ann Martin DuchenneConnect Coordinator & Genetic Counselor

How we help Our family-centered approach is at the heart of everything we do. As your participation grows, so does our ability to strategically invest and navigate in the fight to end Duchenne.

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Accelerating Research The heart and soul of Parent Project Muscular Dystrophy (PPMD) is accelerating Duchenne research.

Since 1994, PPMD has been the leader in the Duchenne field, funding over $45 million directly to Duchenne research and leveraging over $500 million more through government grants and private sources.

BETTER Projects that will improve the quality of drug candidates entereing clinical testing

FASTER

NOW

Projects that will decrease the time required to test drugs for Duchenne and Becker

Reviewing and testing approved drugs to buy time for this generation of boys

Better, Faster, Now 4

The average new drug today costs over $1 billion to

Specifically, PPMD collaborated with Colorado-based,

develop and takes more than ten years – that’s $1 billion

biotechnology company Somalogic and Cincinnati Children’s

the Duchenne community doesn’t have and ten years we

Hospital to collect and analyze blood samples from age-matched

can’t spare. Parent Project Muscular Dystrophy’s research

boys with and without Duchenne, and with and without steroids.

investments focus on what’s next. We have a simple but

Several promising blood markers from these studies have

effective plan: Better, Faster, Now. In each case we set

been identified that may help us monitor the effectiveness of

specific qualitative and quantitative goals that will be

new drugs in faster, less invasive ways than current methods.

measured and reported to the Duchenne community.

PPMD has also contributed significantly to the development We know that drug development is a complicated ecosystem

of magnetic resonance imaging (MRI) at the University of

that benefits from consistent care across centers, valid

Florida as another non-invasive way to measure both loss

regulatory strategies, clinical infrastructure, current natural

of muscle tissue and the potential positive effects of new

history data, availability of biomarkers, and an understanding

treatments. In 2012, PPMD provided funding to the University

of the socioeconomic burden of disease; these are just a few

of Florida investigators to test their MRI methodology on

of the things that can impact the likelihood of success for

subjects participating in Sarepta’s phase II eteplirsen trial

new drugs.

to help validate this technology.

Although we put significant dollars directly into development

Additionally, PPMD launched the Duchenne Drug Development

efforts for particular drugs, this strategy alone will never

Roundtable to bring together representatives of different

move the needle fast enough. We have to enable drug

companies and funding groups to discuss common issues

development efforts across the whole ecosystem in a

in drug development. The first meeting was attended by

holistic manner.

11 different companies developing drugs for Duchenne from small start-up biotechnology companies to major

Enabling Clinical Trials As promising prospects for treatment to move forward, PPMD has been working behind-the-scenes to make sure that drug developers can plan for and recruit for trials as efficiently as possible.

pharmaceutical companies.

Exploring Approved Drugs for Duchenne In addition to funding new therapeutics to treat Duchenne,

PPMD has also undertaken an internal project called RADD

PPMD is also looking at drugs that are already approved

(Review of Approved Drugs for Duchenne), along with

and on the market for other uses that may have some benefit

collaborators at Nationwide Children’s Research Institute,

for Duchenne. This approach has the advantage of being

Newcastle University, and Children’s National Medical Center

immediately available if a particular approved drug is found

to conduct systematic data reviews to better prioritize other

to be effective. To this end, PPMD has funded two promising

approved drugs for further investigation in Duchenne.

clinical trials to test approved drugs, including Tadalafil at

A publication from this group is pending in 2013.

Cedars Sinai in Los Angeles, and Eplerenone at Nationwide Children’s Research Institute.

STRATEGIES: HOW TO FIX THE PROBLEM Changing the DNA

Affecting the RNA

Replacing the Protein

• Gene repair

• Stop codon read-through

• Dystrophin replacement

• Exon skipping

• Other proteins added or stimulated

• Gene therapy

Building New Muscle

• Drugs/gene therapy to stimulate growth • Stem cells

Correcting “downstream” problems

• Improving blood flow • Blocking fibrosis and inflammation • Normalizing calcium

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PPMD’s Funding of the Research Pipeline Our diligence includes: • Financial and management review • Thorough review of the science • S trategic contract negotiations

In 2011 and 2012, PPMD funded an unprecedented number of new experimental therapies for Duchenne. We believe that everyone with Duchenne, no matter what mutation is behind the disease, should be able to benefit from new therapeutics.

Larger drug development projects are milestone-driven with

Key projects include

independent steering committees that oversee completion

• An experimental approach to stimulating new muscle growth by delivering the gene for a protein called follistatin in a modified viral carrier;

of milestones.

Influence & Impact: Partners in Research

• Development by the biotechnology company Tivorsan of a protein fragment called biglycan that can concentrate utrophin at the muscle cell membrane;

We are proud that PPMD leadership serves on many distinguished boards and committees, and partners with many professional associations as part of our mission to end Duchenne.

• Development of an anti-fibrotic compound called HT-100 (halofuginone) by Halo Therapeutics;

• Institute of Medicine (IOM) – Committee on Pediatric Studies

• Reformulation of utrophin-upregulating drug SMT-C1100

by Summit PLC;

• Health Research Alliance (HRA) – Board of Directors • School of Mind, Brain and Behavior, University of Arizona – Advisory Board

• Funding to sites involved in trials of the Sarepta exon 51 skipping drug eteplirsen and the Shire muscle building drug HGT4510 to support participant travel; and

• National Advisory Council on Neurological Disorders and Stroke

• Numerous awards for new, early stage preclinical drug candidates to keep the drug pipeline full.

• Co-chair, HRA working group “Funders of Drug Development”

PPMD has a well-defined process to review these projects,

• TREAT-NMD’s Advisory Committee for Therapeutics, ad hoc member

using its own distinguished Scientific and Drug Development

• Clinical Trials Transformation Initiative Patient Leadership Council

Advisory Committees and also TREAT-NMD’s Advisory

• E xpert Advisory Group for the FasterCures Intellectual Property Intersection Web site – Member

Committee on Therapeutics. We are dedicated to being good stewards of the community’s dollars.

Drug Development Projects funded by PPMD in 2011-2012 Drug

preclinical

phase I

phase II

phase III

Eteplirsen (Sarepta)* Ataluren (PTC) Tadalafil and Sildenafil (Cedars Sinai) Eplerenone (Nationwide and Cincinatti) HGT4510 (Shire)* Follistatin gene therapy (Nationwide Children’s Research Institute) SMT- ­C1100 (Summit) HT-­100 (Halo) Biglycan (Tivorsan) Glycoprotein cardiac treatment (Nationwide) Utrophin up-­regulating drug (U Penn) Multi-­exon skip (University of Alberta) Corticosteroid replacement (CNMC) Tamoxifen (University of Geneva) SERCA2A gene therapy for cardiomyopathy (U Missouri) *Grants made directly to clinical sites to help cover participant travel costs

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Advocacy in Action In the fight to end Duchenne, one of the most powerful tools we have as a community is our voice. Parent Project Muscular Dystrophy has been leading this fight in Washington, D.C. for over a decade – informing decision makers in Congress, demanding recognition from federal regulatory agencies, and encouraging families to share their stories from the local level all the way to Capitol Hill.

Reauthorizing the MD-CARE Act

and Representatives introduced the MD-CARE Act

A pivotal moment in the history of Duchenne occurred when

Amendments of 2013. This legislation seeks to update

The Muscular Dystrophy Community Assistance, Research, and

the landmark MD-CARE Act law. We have every confidence

Education (MD-CARE) Act was signed into law by President

that Congress will continue to support this critical legislation,

George W. Bush in December of 2001. This was the first

which helps fund vital research and continues to give hope

legislation in the history of the US Congress that focused on

to families.

muscular dystrophy and PPMD is enormously proud of our leadership role in getting this legislation passed. The bipartisan MD-CARE Act provides important authority and direction for muscular dystrophy research, including Duchenne.

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This legislation included 4 major points: • NIH would support Centers of Excellence focused on muscular dystrophy.

• CDC would establish programs focused on Duchenne muscular dystrophy.

• NIH and related government agencies would convene the research and clinical community to develop a research plan.

Our Relationship with the FDA PPMD is proud of the relationship we have built over the last decade with the Food and Drug Administration (FDA). We continue to work with agency leaders to help strike a balance between clinical certainty and patient access to potentially life-saving drugs.

In 2011, PPMD’s Board of Directors approved and submitted our official FDA Policy to identify the needs and barriers for developing clinical trials.

• NIH and related government agencies would establish a steering committee to oversee progress (MDCC).

In late 2012, we launched the most ambitious Duchenne

In 2008, the MD-CARE Act was reauthorized due in large

Therapies program. This program is intended to inform the

part to the leadership and commitment of our advocacy campaign. In February 2013, a bipartisan group of Senators

PPMD’s 2013 Advocacy Conference

162

170

3,764

Advocates attending the Advocacy Conference

Face to face meetings with Congress

Packet drop off letters delivered

428

/435 Districts

50

/50 States

FDA, industry, and the community about the risk tolerance, benefit preferences, and treatment priorities of the Duchenne community. There are several parts to the program, including a recently-completed treatment survey, the data of which is currently being analyzed and compiled for eventual publication, and a “share your story” opportunity that ask

THE NUMBERS

COVERING

advocacy strategy to date: PPMD’s Benefit/Risk in Duchenne

families to describe their Duchenne experience. Data collected from all parts of this program will be shared with the FDA. As 2013 moves ahead, so does PPMD’s commitment to increasing our engagement with the FDA. Now that the landmark Food and Drug Administration Safety and Innovation Act (FDASIA) legislation, that includes the Prescription Drug User Fee Act (PDUFA), has passed, PPMD will provide the FDA with recommendations to speed responsible access to new therapies for Duchenne and other rare, serious, and life-threatening disorders.

Putting Patients First: Recommendations to speed responsible

In 2011, PPMD hosted the One Voice Summit. This meeting

access to new therapies for Duchenne muscular dystrophy

analyzed the progress of care and research in Duchenne since

and other rare, serious and life-threatening neurologic

the passing of the MD-CARE Act in 2001. From this Summit,

disorders is a white paper that hopes to ensure that the FDA

we published a report with findings to inform the next update

makes the most of these new opportunities under FDASIA.

to the MD Action Plan developed by the Muscular Dystrophy

With the help of an expert Advisory Committee (including

Coordinating Committee (as required by the 2001 bill).

leading voices in academia, industry, and patient advocacy), PPMD has outlined how the FDA can effectively evaluate new

Year round, PPMD calls on the Duchenne community to

therapies. We are pleased to share these recommendations

advocate from home. Writing, emailing, calling, and even

with the community and begin to disseminate them to all

visiting your representatives in district – each helps to

stakeholders. The complete white paper can be found at

carry our message and our stories to the decision makers

ParentProjectMD.org/PuttingPatientsFirst.

in Washington. We encourage families and friends of the community to sign up for these regular calls to action and

We will continue to update and adapt PPMD’s advocacy

lend their voice to ours.

agenda as the climate in Washington changes because we are in this for the long haul. It’s not about one therapy or one trial—it’s about making the drug approval process work better to benefit our Duchenne community.

Influence & Impact: Partners in Advocacy We are proud that PPMD leadership serves on many distinguished boards and committees, and partners with many professional associations as part of our mission to

Advocacy in Action

end Duchenne.

Our extensive advocacy agenda is successful because our

• D epartment of Defense (DOD) – Chair of Congressionally Directed Medical Research Program in Duchenne

community supports it. Dedicated families and friends of people with Duchenne know that their voices are critical in Washington so that Duchenne research and care can continue to get much needed funding from the government.

One way families show their support is by attending PPMD’s Annual Advocacy Conference each February in Washington, D.C. The Annual Advocacy Conference is the only officially organized gathering on Capitol Hill for the Duchenne community. Each year, participation grows with more and more congressional districts represented. Scheduled visits on Capitol Hill are arranged for all attendees, giving a face and a voice to PPMD’s advocacy agenda.

• National

Organization for Rare Disorders (NORD) – Board of Directors • Alliance for a Stronger FDA • Rare Disease Legislative Advocates (RDLA)

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Optimizing Care Perhaps the greatest advance that has been made in the fight to end Duchenne over the last years has been in the way we care for those diagnosed with Duchenne. Parent Project Muscular Dystrophy takes its role as a leader in the Duchenne community very seriously, and insisted that standards of care be put in place.

The result is that people with Duchenne are living longer, healthier, happier lives.

Understanding Gaps in Care

the country. The webinars described cardiac observation,

Since 2006, PPMD has worked diligently to enhance the care

screening, medical management, and mechanical interventions

of patients and families living with Duchenne. We brought

for both patients and carriers. The webinar received accolades

together medical experts, researchers, parents, and patients

from both the private and professional sectors. The webinars

in order to address areas of need and gaps in care. These

have been archived and are available online, and continue to be

areas include cardiac care, learning and behavior, adjusting

viewed by parents, patients, and providers around the world.

to the diagnosis, endocrine, and metabolism. The discussions from these meetings have been reported to the community

Over the past several years, PPMD has heavily invested in the pursuit of optimal care, interventions, and research pertaining to the management and prevention of cardiomyopathy/heart failure. In 2011, PPMD funded nearly $2 million toward developing a comprehensive Cardiac Initiative. PPMD continues to advocate for novel and ongoing research, as well optimal medical and mechanical

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cardiac management, and have committed an additional $1 million in 2013/14 to this effort.

To understand and improve all aspects of Duchenne care in the United Sates, PPMD launched the Transforming Duchenne Care initiative in June 2012. Starting with a twoday meeting prior to the 2012 Connect Conference in Fort Lauderdale, Transforming Duchenne Care brought together leadership from top medical institutions, neuromuscular experts and thought leaders, colleagues from the MDA, and patient representatives to discuss, in an open forum, the ideal configuration, features, services, and structure of stateof-the-art family centered care for patients with Duchenne. and help inform updates to the Care Considerations, which

The success of this first meeting led to the “Transforming

were published in 2009/2010. Improvements in medical

Duchenne Care Initiative,” a two-phase initiative addressing

care have extended the life span of individuals living with

transparency in clinical services and care. The Initiative’s

Duchenne, and many of these elements of care are especially

Core Team embraced providing more information to patients

important as our Duchenne population continues to age.

and families about care provided at individual clinics. We are pleased that the MDA has recently added more information

PPMD has an ongoing commitment to cardiac care. Heart

about care capabilities of their MDA-supported clinics to their

issues will affect every person living with Duchenne, and

website. The Transforming Duchenne Care Core Team continues

dilated cardiomyopathy/heart failure remains the leading

to work to provide information about care capabilities at

cause of death in Duchenne. The “Cardiac Webinar Series”

all clinics, as well beginning the process of evaluating care

connected clinicians, families, and patients with top

and exploring clinic accreditation. This exciting effort will

cardiologists and cardiothoracic surgeons from around

continue into the coming year.

PPMD’s Care Resources

In Fall of 2012, we launched a web-based diagnostic tool

PPMD takes pride in being your go-to resource for care

(www.childmuscleweakness.org) to help healthcare providers

issues and concerns. We are constantly updating our website,

notice symptoms of weakness sooner and make appropriate

app, and handouts, so that you are armed with up-to-date

referrals. The web tool includes a motor surveillance aid and

information at the critical moment you may need it.

motor delay algorithm for testing and referral. It also includes an extensive video library highlighting early and later signs of

Our wallet-sized Emergency Care Card is one of PPMD’s

muscle weakness compared to normal motor development.

most popular resources. It is likely that if an emergency

The website has been highlighted on Medcape, the CDC

occurs, patients will be seen by professionals who have no

website, and in new American Academy of Pediatrics guidelines.

knowledge of them or of their disease. We have developed a simple, yet extremely important card containing information

Influence & Impact: Partners in Care

pertinent to the emergency care of a sick or injured patient

We are proud that PPMD leadership serves on many

with Duchenne. This resource is constantly being updated.

distinguished boards and committees, and partners with

Specific issues regarding the emergency use of oxygen

many professional associations as part of our mission to

emerged and were addressed this year, and the “respiratory

end Duchenne.

care” section of the card was updated and refined. The

• American Council for Genetic Counseling – Past President

Emergency Care Card is free and can be ordered on our website.

In 2012, PPMD was pleased to launch new and improved Duchenne Care Fact Sheets, which contain recommendations consistent with the Care Considerations. These handouts are complementary to our revised and updated care section on our website, and are available online. Fact sheets are available for corticosteroid use, muscle function, heart care, bone health, GI concerns, lung care, and psychosocial concerns.

By Summer 2013, PPMD will unveil our revised and updated care section on our website. The care materials are provided in a user-friendly format, allowing users to choose topics and the depth of detail with greater ease. Parent advisors guided the update of our care materials. We intend the care pages to be a resource that grow and develop over time.

In late 2013 and into 2014, we will add additional materials that include care sheets for Becker muscular dystrophy, specific information for fathers and grandparents, and more information on heart care.

Early Identification and the Child Muscle Weakness project Through a cooperative agreement with the CDC, PPMD lead The National Task Force for Early Identification of Childhood Neuromuscular Disorders to assist healthcare providers in the evaluation of children with motor weakness and delay.

• M ember of the Office of Rare Disease Research Global Rare Diseases (Patient) Registry and Data Repository (GRDR) selection committee in 2012 • C DC Working Group on Quality of Life Models and Measures for People with Muscular Dystrophy and Other Significant Mobility Limitations • P PMD staff and consulting professionals now include three experienced healthcare providers: 2 board-certified genetic counselors and a nurse practitioner

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Providing Education and Community Resources Educating the global Duchenne community is an important part of Parent Project Muscular Dystrophy’s mission. We know that in rare disease it is easy to feel uninformed and isolated. PPMD strives to connect the Duchenne space and unite the community.

DuchenneConnect Registry

into neuromuscular clinics. In 2013 new registry elements will

DuchenneConnect.org is a robust and cutting-edge registry

be available, including a more interactive individualized page

and resource that connects people with Duchenne and

for each registrant.

Becker muscular dystrophy with actively recruiting clinical families about research. At the same time, DuchenneConnect

Annual Connect Conference and Educational Series

is a valuable resource for researchers and companies,

The Annual Connect Conference started almost two decades

allowing access to de-identified (anonymous) information

ago as a small group of parents gathering to discuss current

provided by patients and their families—information that

Duchenne topics. It has evolved into the largest, most

is vital to advances in the care and treatment of Duchenne.

comprehensive annual, international conference focused

The DuchenneConnect coordinator is available to answer

entirely on Duchenne. In 2012, we expanded our Connect

questions about genetic testing and clinical trials for patients

series to include an additional West Coast Meeting in San Diego.

trials and research studies, and educates patients and

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and families.

Our meetings are 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.

Expectations and Perceptions of Clinical Trials Profile Survey Provide researchers with data to help develop new clinical trials for Duchenne and find out if you are eligible for upcoming studies.

Clinical Services Do you wonder how your clinic or doctor compares to others who care for Duchenne? Help us spread the word about clinical care.

Explore Data See how you or your child compare to others in the registry. Explore the newest information from more than 2,000 participants.

In 2011, PPMD was awarded an NINDS-funded grant to evaluate expectations and experiences in Duchenne and Becker muscular dystrophy clinical trials. The overall goal of the program is to find targets to improve the well-being of families considering or involved in clinical trials. The program is lead by a diverse advisory committee that includes adults with Duchenne, parents of people with Duchenne, a bio-ethicist, a neurologist, a translational researcher, and social science

In 2012, there were 405 new patient registrants and a total of

researchers.

2,187 completed Profile Surveys. An updated Profile Survey went live in June 2012, with the goal of obtaining a more

In 2012, PPMD completed interviews with parents of children

robust dataset containing the key elements needed by both

who were in Duchenne/Becker clinical trials, clinicians

industry and academic researchers. DuchenneConnect was

involved with clinical trials, and translational researchers

used to recruit for seven clinical trials in 2012 (85% of all trials

working on animal models of Duchenne/Becker muscular

recruiting in 2012) and for five research studies. We are also

dystrophy; in addition, we conducted a focus group of

working to improve the completeness and diversity of the registry

individuals with Duchenne who participated in clinical trials.

through a pilot project where we put DuchenneConnect tablets

We plan additional interviews and several surveys in 2013.

Connecting and Educating the Community

Influence and Impact: Partners in Education

PPMD serves a community with varying levels of connectivity.

We are proud that PPMD leadership serves on many

For this reason, we are very careful to understand and take

distinguished boards and committees, and partners with

advantage of the numerous ways in which to communicate.

many professional associations as part of our mission to end Duchenne.

PPMD’s website, www.ParentProjectMD.org is the go to resource for the Duchenne community, posting the latest announcements, but also maintaining rich and up-to-date content on all aspects of living with Duchenne.

In addition to our main site, PPMD boasts the largest Duchenne-specific community site (community. parentprojectmd.org). With over 4,000 members, this site offers families across the world access to each other in a social media style environment dedicated strictly to the world of Duchenne.

In 2011, PPMD introduced the first Duchenne-specific mobile application. Now news updates are available in a mobile format and soon a second app, the Duchenne Central app, will be released to help the Duchenne community navigate treatments and trials from the palm of their hand.

As social media has grown over the past two years, so has PPMD’s presence on social media. Now boasting over 13,000 Facebook likes and 1,500 Twitter followers, PPMD’s reach and ability to share information continues to broaden.

• E xecutive Committee member for the Johns Hopkins/ National Institutes of Health Genetic Counseling Training Program • S ubject Matter Expert/Reviewer, American Academy of Pediatrics PediaLink Early Detection of Neuromotor Disorders course • P PMD staff regularly provides trainings for healthcare providers, educators, researchers, families, and other stakeholders.

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Funding Hope Nothing is more powerful than a community coming together for a common cause. Parent Project Muscular Dystrophy’s is successful thanks to parent-led passion, dedication from this community, and the efforts of many. This community is our strongest weapon in the fight to end Duchenne. You are the driving force behind all that we do.

Run for Our Sons Run For Our Sons is PPMD’s endurance sports program

Since 2005, nearly 3,000 runners have worn the Run For Our

aimed at creating awareness and raising funds for Duchenne.

Sons team name and have helped to raise over $7 million.

The funds raised from all Run For Our Sons events continue

Run For Our Sons teams and fans participate in major

to push PPMD’s programs forward, and every step taken and

marathons and half marathons around the country and

every dollar raised is critical in our fight to end Duchenne.

inspire crowds to go the distance to end Duchenne. In towns

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and cities all over America, families, friends, churches, and

Coach to Cure MD

schools host family fun runs, 5Ks, and 10Ks to raise awareness

Coach To Cure MD is a partnership between the American

of Duchenne. Our website, RunForOurSons.org, makes it

Football Coaches Association (AFCA), a professional

easy for runners, “spirit” runners, families, and friends to

organization for over 12,000 college football coaches

go the distance for Duchenne research and treatment.

and staff, and PPMD.

In 2012, Run For Our Sons raised more than $1.3 million –

In 2008, the AFCA adopted PPMD’s Coach To Cure MD

a record-breaking year for our endurance program. Run

program as one of their charity efforts. One reason the AFCA

For Our Sons began nine years ago, with one race and

was drawn to Coach To Cure MD was because of the unique

a determined group of runners at Disney World. Today,

parallels between Duchenne, a disorder which robs young

we have expanded into 15+ races across the country and

men of precious muscle strength and college football, a game

thousands of participants. We added new races last year in

where young men are at the peak of their muscle strength.

Baltimore, New Orleans, Cleveland, and Grand Rapids, and expanded our program to include the prestigious New York

The goals of Coach To Cure MD are simple:

City Triathlon and Conquer the Canyon, a Grand Canyon

• Raise national awareness of the disorder

hiking adventure. This incredible year was all thanks to our

• Raise money to fund critical Duchenne research

runners and cheerleaders!

By wearing armbands, mentioning Coach To Cure MD during

PPMD’s monthly giving campaign called STIR (Striving

on and off-field interviews, and in some instances doing more

To Impact Research) is an easy and effective way for small

extensive media relations around the event, coaches are

donations to cumulatively add to a larger sum. This program

rolling up their sleeves and proudly getting involved.

has raised over $1 million for research over the years and continues to be an important source of funding for our

The 2012 season marked the fifth year that college football

research programs.

coaches nationwide joined together in support of the Coach To Cure MD program, which is held the last weekend of

PPMD’s Text-To-Give program is an exciting and new way

September. In 2012, more than 11,000 college coaches at

to raise funds. Donors simply text the word “CURE” to

585 different institutions participated in the Coach To Cure

90999 and a donation of $5 is made to PPMD. This kind

MD event. The rapidly growing annual effort has raised over

of fundraising is a great addition to events and is an easy

a million dollars to date.

add-on for all of our fundraising programs to include.

In addition to the coaches’ efforts, families affected with

In 2011, PPMD introduced Shop For Our Sons, a partnership

Duchenne muscular dystrophy will also gather for fundraising

with Amazon whereby PPMD is given a percentage of sales

tailgate parties on campuses around the country to

when shoppers use our dedicated PPMD link to Amazon.

encourage more fans to get involved.

This program continues to thrive and is an easy way for our families and friends to help boost fundraising potential.

The Power of Grassroots Efforts PPMD’s Grassroots efforts include a variety of different ways

In addition to all of the above mentioned programs, PPMD

in which families, friends, sponsors, and other foundations

also has a strong Matching Gifts program, participates in

can roll up their sleeves and get involved with us. Our

many of the United Way campaigns across the country, and

organization started out through the support of grassroots

is a member of the Combined Federal Campaign.

events, and today these family-led fundraising events continue to be a huge driving force of our work. In 2012,

Fundraising is not easy, but it is an important part of how

PPMD assisted families across the country with over 45

PPMD is able to accomplish our mission. Becoming involved

different events, which raised just over $800,000!

in any of the many ways in which PPMD raises funds not only helps us reach our goals, it empowers the families and friends

PPMD also recognizes that hosting an event can be quite

involved and helps them to know they are an active part of

time consuming and not a perfect fit for everyone. Our other

ending Duchenne.

grassroots fundraising efforts are aimed to offer everyone in our community a level of comfort.

15

16

What’s Next

Influence and Impact: Partners in Fundraising

In addition to the many fundraising programs and grassroots

We are proud that PPMD leadership serves on many

efforts, PPMD’s major donor and corporate sponsorship

distinguished boards and committees, and partners with

programs continue to be a cornerstone of our funding

many professional associations as part of our mission to

efforts. In 2012, PPMD‘s What’s Next major gift campaign led

end Duchenne.

our research funding with over $2.5 million in contributions.

One of the most incredible elements of the What’s Next campaign was the first End Duchenne Gala which was held in New York City in early December. Led by a dedicated team of PPMD Board Members and Duchenne community leaders, the End Duchenne Gala raised over $300,000 its first year.

• Better Business Bureau’s Wise Giving Alliance • Charity Navigator – 4 star rating • Combined Federal Campaign • United Way • A ssociation of Fundraising Professionals • Run Walk Ride Council • American Football Coaches Association

In 2013, PPMD will continue to grow our major giving and corporate programs to keep up with the ever growing funding requests for research and clinical trials. We are excited about the evolution of What’s Next and about introducing new tools in the management of this important campaign.

2012 Corporate Sponsors Advance Digital

Nationwide Children’s

Samuel I. Newhouse Foundation Inc.

Catabasis

Pfizer

Shire

Goldman Sachs

Philips Respironics

Summit PLC

IPC International Corporation

PTC Therapeutics

Tivorsan

Masters

Sarepta Therapeutics

Volox Motion Inc.

MLIVE

2012 Statement of Activities

2012 2011

Unrestricted net assets

Revenue and other support Grants and contributions

3,274,324

5,195,537

Program revenue

52,208

77,571

Investment loss

(8,298)

(9,469)

2,310

9,589

1,222,740

1,059,625

4,543,284

6,332,853

5,135,095

5,216,305

Management and general expenses

415,712

417,787

Fundraising expenses

672,392

585,946

6,223,199

6,220,038

(1,679,915)

112,815

Interest and dividend income Fundraising proceeds (net of direct expenses of $365,921 and $333,208, respectively)

Expenses Program services

Change in unrestricted net assets

Temporarily restricted net assets Restricted contributions

1,237,485

-

Increase in temporarily restricted net assets

1,237,485

-

Increase (decrease) in net assets

(442,430)

112,815

Net assets - beginning of period

2,848,336

2,735,521

Net assets - end of period

2,405,906

2,848,336

See audited financials on ParentProjectMD.org

Today we are closer than ever to ending Duchenne.

With your help, your support, and your actions you can make this possible.

Visit ParentProjectMD.org/Possible.

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