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.
401 Hackensack Avenue, 9th Floor Hackensack, NJ 07601 • T. 800.714.5437