PROGRESS REPORT
COMMUNITY
ONLINE
S.T.I.R.
REGIONAL ROUNDTABLE
PROJECT CATALYST
DUCHENNE CONNECT
EDUCATION
MD-CARE ACT
CARE
RUN FOR OUR SONS
RESEARCH HOPE UNITY
ADVOCACY
LEADING PTC-124 THE FIGHT ANNUAL CONFERENCE TO END FAMILY DUCHENNE CORPORATE SPONSORSHIPS
SUPPORT
DEAR PPMD FAMILY
Dear PPMD Family, As we look to the future, we see promise and hope where there used to be very little. For the first time ever, there is a strong belief within the Duchenne research community that boys of this generation will not only become adults but also live out their dreams. We had no such hope when my sons were diagnosed. The entire Duchenne community is focused on accelerating the development of treatments so that all boys diagnosed have access to treatments that will stop their progression of Duchenne. Exciting new options are being moved into clinical trial, and research is advancing faster than ever before. We’re seeing an exciting level of new cooperation and collaboration in the Duchenne research field, and we’ve also worked toward our shared advocacy goals. In addition to research and advocacy, the PPMD staff works hard to advance care and treatment for families coping with Duchenne today. And we continue to raise awareness beyond the Duchenne community and educate the public in order to reach families who haven’t yet had a Duchenne diagnosis. It is Parent Project Muscular Dystrophy’s comprehensive approach in the fight that will end Duchenne. We must end Duchenne. And we will end Duchenne. Sincerely,
Pat Furlong Founding President & CEO Parent Project Muscular Dystrophy
Dear Friends, Families, and Supporters, As the father of a boy with Duchenne, I’ve got many personal, emotional reasons to do everything I can to help Parent Project Muscular Dystrophy end Duchenne. I want to see my son walk farther, climb higher, and live longer. It’s a matter of life and death for my family. As a businessman, I’ve also got logical, professional reasons to help PPMD end Duchenne. PPMD is the only Duchenne organization to invest in a broad research portfolio, including short-, mid-, and longterm treatments and therapies. No other organization has leveraged hundreds of millions of dollars into Duchenne-specific programs and research centers. They are the only muscular dystrophy organization to receive a seal-of-approval from the Better Business Bureau’s Wise Giving Alliance and multiple four-star ratings from Charity Navigator. Parent Project Muscular Dystrophy is a leader among muscular dystrophy organizations. You can’t make a better, wiser investment. It is our duty to do everything we can to help end Duchenne: raise awareness, join a Run For Our Sons team, advocate in DC, make donations large and small, and more. Because no other organization works so tirelessly and effectively in the fight against Duchenne, we must also work tirelessly to support Parent Project Muscular Dystrophy. I hope you’ll join me in these efforts. Thank you. Best,
John Killian Board Chair Parent Project Muscular Dystrophy
FRIENDS
DEAR
DUCHENNE
Duchenne muscular dystrophy (Duchenne) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (about 20,000 new cases each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures. Duchenne can be passed from parent to child, but approximately 35% of cases occur because of a random spontaneous mutation. In other words, Duchenne can affect any family. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne, and young men with Duchenne typically live only into their late twenties.
Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy. We are the only Duchenne organization • To invest in a broad research portfolio; • To explore short-, mid-, and longterm therapies; •T o leverage $200 million into research and centers and $22 million into Duchenne-specific programs at the Centers for Disease Control
• To leverage $15.4 million from the National Institutes of Health into a drug discovery program; • To receive a seal-of-approval from the Better Business Bureau’s Wise Giving Alliance; • To receive four stars from Charity Navigator several years running.
and Prevention; Since 1994, PPMD has improved the treatment, quality of life, and long-term outlook for all individuals affected by Duchenne through research, advocacy, care, and education. Because of our efforts, families affected by Duchenne have better access to state-of-the-art care information, research is moving forward at an accelerated pace, and legislation now exists to fund Duchenne research and outreach programs. The name of the organization reflects our grassroots origins, parent-led focus, and passion, and Parent Project Muscular Dystrophy is now a name recognized around the world as the leader in the Duchenne community. We hold the highest ethical standards and consistently receive high marks from watchdog organizations. We take a comprehensive approach in the fight against Duchenne— funding research, promoting advocacy, connecting the community, broadening treatment options, and raising awareness. Only this comprehensive approach will lead to the day that 100% of those diagnosed can turn to a treatment that will end Duchenne muscular dystrophy.
PROJECT DYSTROPHY
PARENT MUSCULAR
RECENT ACHIEVEM • Invested in research and potential treatments that will affect the entire Duchenne community • Accelerated the first-ever drug treatment and clinical trial for Duchenne, developing a path for future therapies • Signed a $1 million agreement with PTC Therapeutics for a cardiac target, to fight the primary cause of death for all boys with Duchenne (heart failure) • Developed DuchenneConnect.org, a clinical trials and treatment resource community for families, healthcare professionals, researchers, industry, and policymakers (in English and Spanish)
• Launched expanded PPMD website, ParentProjectMD.org, with deeper content for those affected by Duchenne, those who are newly diagnosed or seeking more basic information, and those who want to be more deeply involved with PPMD • Developed 15-, 30-, and 60-second public service announcements (PSAs) featuring Olympia Dukakis, and distributed them to a national network of television and radio stations that are currently running them pro bono • Hosted our annual conference, which continues to see a 20% growth each year, with over 50 scientific and medical professionals and 480 family members attending in 2007
RESEARCH
When we began our work, the Duchenne research field was virtually nonexistent. We were the first organization to initiate a drug-treatment focused initiative to invest in this generation of boys affected by Duchenne. And because of our investment the field is now ripe with promise. We will continue to fund a broad research portfolio—including short-, mid-, and longterm treatment and therapies—until every patient with Duchenne can turn to solution that will stop the progression of the disease and save his life.
Our broad approach PTC-124 offers terrific promise for about 15% of boys with Duchenne, and we are thrilled that it’s now in clinical trials. But our work doesn’t stop there. What about the other 85%? We’ve invested in numerous promising research projects to combat the many root causes of Duchenne: • Protease inhibitors, which slow muscle degeneration • Compounds that improve blood flow to muscles • Anti-inflammatories to lessen inflammation in degenerating muscles
• Antisense Oligonucleotide (AON) drugs, which produce a shortened but efficient protein to replace Dystrophin • Proteins (including Utrophin and Integrin) that can replace Dystrophin in muscles
MENTS • Launched pilot outreach program in Mississippi in partnership with the Centers for Disease Control and Prevention to
ParentProjectMD.org, while broadly raising awareness for Duchenne at no cost • Continued to expand Regional Roundtables,
º Raise awareness and reach out to families who may not yet have received a Duchenne
providing up-to-date information on Duchenne
diagnosis, and those who may be receiving
“Virtual Roundtable,” which reaches a broader
inadequate care
audience that simply logs in online to listen to
º Educate healthcare professionals so that they recognize the signs and lessen the diagnostic odyssey for parents • Launched banner advertising campaign with a media firm working pro bono, which has served up millions of Duchenne-specific awareness impressions online, resulting in increased traffic
research and care, including the first-ever online
the presenter • Offered online seminars for professionals in identifying muscle weakness, with particular emphasis on Duchenne identification and diagnosis • Led workshops targeted on gaps in knowledge about care, including cardiac and behavior issues
to both DuchenneConnect.org, and
• Compounds that regulate muscle size and strength • Compounds that block calcium overload in muscle cells In the past few years, PPMD has invested over $7 million dollars in Duchenne-specific research, aimed at helping this and future generations of young men live longer, healthier lives.
Researching to End Duchenne Melissa Spencer, Professor of Neurology at UCLA, has been a longtime fan of PPMD. “PPMD provided the early funding that allows us to do a pilot study that, in turn, led to other funding.” For Melissa, PPMD’s impact is direct and local. “One of the most important things PPMD does is to fund small research projects,” such as the $40,000 that Melissa’s department received. “It’s a Catch-22, because you can’t get an NIH grant without preliminary data, and you can’t get data without funding. PPMD allows investigators to explore promising avenues of research that may not yet be ready for NIH-level funding. PPMD helps to bridge that gap.” Melissa also appreciates what she sees as PPMD’s global influence. “There are now muscular dystrophy parent groups across Europe, modeled upon PPMD’s approach, that translate and disseminate PPMD’s findings in their own countries. That’s a powerful demonstration of PPMD’s effect on the global conversation.”
WHAT WE ARE FUNDING
RESEARCH INITIATIVE
Funding
Project Catalyst: PTC Therapeutics Drug Development High throughput screens (HTS) (Utrophin, Alpha7Integrin, Myostatin inhibition, IGF-1)
$2.6 million (2005-2008)
Catalyst PTC Therapeutics Cardiac Specific Target (Serca 2a)
$250,000 HTS (2008-2009); $750,000 pending results/lead optimization
Andrew Hoey, U. Southern Queensland Upregulation of nNos to slow muscle degeneration using approved drugs; Investigation of Pirfenidone to improve cardiac function
$100,000 per year for three years (2008 will be year three)
Large Animal Model, U. Missouri Investigation antisense oligonucleotides and viral gene therapy (Koregay)
$264,946 (2007)
Large Animal Model, U. Pennsylvania Meg Sleeper, Therapeutics in Duchenne
$66,000 (2008)
Dominic Wells, Imperial College Antisense Oligonucleotides Dosing studies
$100,000 (2007-2008)
Brian Tseng, U. Colorado (currently Mass General Hospital/Boston) Investigate potential benefit of Protandim on free radicals and fibrosis
$20,000 (2007)
Melissa Spencer, UCLA, Fellowship support; Immune response to muscle cell injury
$40,000 (2007)
Rob Kotin AAV production for viral gene therapy
$50,000 (2007)
Beth Barton, U. Pennsylvania IGF-1 Isoforms or Iplex to improve functional mass in animal models for Duchenne
$100,000 per year for two years (2007 and 2008)
Bowman Birk Inhibitor Compound Investigate potential to slow/halt muscle degeneration; Explore patent issues related to compound
$140,000 (2007)
Justin Fallon Byglycan
$65,000 (2007); $65,000 pending (2008)
Antisense oligonucleotides Feasibility study Investigate application of AON in Duchenne for individuals with duplications/large deletions
$500,000 pending (2008)
Krista Vandenborne, U. Florida/Gainesville MRI study to evaluate MRI as a potential outcome measure in Duchenne
$30,000 (2007) $96,000 (2008)
Fellowships: Peter B. Weisman Fellowship
$50,000 (2007)
DuchenneConnect Patient self-report registry; Genotyping campaign with Emory Genetics (Atlanta) to facilitate genetic testing, and data collection to facilitate participation in upcoming clinical trials
$250,000 (2007) $500,000 (2008)
Interdisciplinary Care Model, Cincinnati Children’s Hospital
Pending
Ethnic Diversity Understand racial disparity; Determine if disparity is the result of access to care or a biological question
$10,000 (2007)
Ongoing Legislative Agenda MD-CARE Act 2008, Reauthorization of MD-CARE Act
$300,000 per year (2000-2008); Leveraged $200 million for muscular dystrophy, and $50 million specifically for Duchenne
DuchenneConnect Advances in genetic testing offer the opportunity to understand each child’s specific genetic mutation (that is, what happened to the gene that resulted in Duchenne). The challenge remains to collect the data necessary to understand the relationship between the genotype (genetic mutation) and the phenotype (clinical course). In response to this challenge, PPMD launched DuchenneConnect.org, one of our most significant recent contributions to the Duchenne community. This revolutionary new approach to the clinical trial process was created to serve as a central hub linking the resources and needs of the Duchenne/Becker muscular dystrophy community: young men with Duchenne; their families and caregivers; and the medical and research provider community. DuchenneConnect provides an opportunity for families/patients to report on their various conditions. In turn, it offers researchers/industry the chance to better understand mutation and progression so that they can develop novel ways to treat Duchenne. DuchenneConnect.org is interactive, notifying families of promising clinical trials, and offers researchers/industry the opportunity to understand the course of the disorder and patient population, thereby accelerating the development of new treatments.
DuchenneConnect: Early Success The early response to DuchenneConnect has been remarkable, reinforcing our belief that this online resource meets a very real need within the clinical trial process. In fact, in its first six months online, over 1,000 individuals registered with DuchenneConnect.org. As a measure of DuchenneConnect’s global reach, registrants reside in 53 countries and speak many languages, including English, Spanish, French, German, Italian, Chinese, and Vietnamese.
AND TRIALS
TREATMENT
TREATMENT
AND TRIALS Induction of Giovanna Spinella Creating a standard
program director for that office. Dr. Spinella
of care for Duchenne
is a resident of Falls Church, Virginia, and
patients has been a
until recently served as staff neurologist at
long-standing need
Walter Reed Army Medical Center.
in our community. In 2007 PPMD hired Dr. Giovanna M. Spinella as its medical director to provide an all-important objective, expert voice to help guide the process of creating the standard.
“Trying to create programs where one can offer the highest quality clinical care is a challenging and exciting opportunity,” said Dr. Spinella. “I think we can raise the bar on the care, health, and quality of life for the boys with Duchenne. Because care for these boys is now irregular and inconsistent,
Dr. Spinella is a pediatric neurologist with
a standard approach is needed to get
16 years of experience at the National
accurate data.”
Institutes of Health (NIH), the country’s leading medical research agency. She is a consultant for the Office of Rare Diseases at the NIH and spent four years as the science
We look forward to working with Dr. Spinella in improving the treatment and care of boys with Duchenne.
Cincinnati Children’s Hospital Because treatment for Duchenne is so complicated, patients and their families have traditionally juggled appointments at many specialists’ offices, usually in many locations. And then, often, there’s very little communication between care providers. Parent Project Muscular Dystrophy has worked with Cincinnati Children’s Hospital (CCH) over several years to create a new model for care. Families travel to Cincinnati, and CCH puts them up in a hotel for a few days. Before they arrive, a concierge organizes appointments with the many specialists the family needs to see during their visit. The team of specialists works together to assess the needs of each patient, and they stay in contact with the patient’s primary care physician in his hometown. The treatment for Duchenne at CCH has generated interest from hospitals in other cities— including Los Angeles, Boston, Philadelphia, Denver, and Kansas City—which hope to duplicate CCH’s excellent interdisciplinary care model.
Connecting to End Duchenne John and Kristin Hiatt
“If we could clone Dr. Wong and put her
knew something was
(and CCH’s standards of care) in the Chicago
wrong with their son
hospital system, it would save us a very long
Liam. The symptoms
commute,” says John, “but it’s well worth the
led them to suspect
trip. Their approach is comprehensive and
Duchenne, but doctors
proactive. For example, we now have a
in Chicago hospitals
cough assist machine to help keep Liam’s
told them it was
lungs clear. If we’d followed our Chicago
simple motor delay. Unconvinced, they hit the
doctors’ advice, we would have waited until
Internet and discovered PPMD’s website.
he developed pneumonia.”
Upon recommendations from Pat Furlong
The Hiatts are also registered members
(and other parents) they contacted Dr. Brenda
of DuchenneConnect. “Other MD databases
Wong, Doctor of Neurology at Cincinnati
confuse potential registrants with issues
Children’s Hospital Medical Center, and
of eligibility and present great difficulty in
made the four-hour drive from the Chicago
getting quick answers to questions.
area for testing.
DuchenneConnect is set up clearly and is easier to use and understand.”
ADVOCACY
Parent Project Muscular Dystrophy has facilitated interactions between families and members of Congress for years through the annual Advocacy Conference in Washington, DC, legislative roundtables, and more. Our efforts have secured tens of millions of dollars toward Duchenne research and care centers from the Centers for Disease Control (CDC) and Prevention and the National Institutes of Health (NIH).
Advocacy stats • In 2001, Congress passed the MD-Care
• PPMD’s advocates visit hundreds of
Act, the first legislation for muscular
congressional offices annually urging
dystrophy. It is also the only condition-
an increase in funding at the National
specific legislation to become law in
Institutes of Health and the Centers for
recent years
Disease Control and Prevention
• Since passage of MD-Care Act, more
• Pat Furlong is a board member of the
than $200 million has been invested in
MDCC (Muscular Dystrophy Coordinating
muscular dystrophy research with more
Committee), and a committee member
than $45 million going to Duchenne-
on the Collaboration in Education and
specific research
Test Translation Program
• A total of six Wellstone Centers of
• Pat Furlong also serves on the data
Excellence have been established
safety monitoring board for both the
because of MD-Care Act
Rare Diseases Clinical Research Network
• PPMD’s annual Advocacy Conference unites hundreds of families from across
and Cooperative International Neuromuscular Research Group
the United States and is the only organized Duchenne advocacy event
The MD-Care Act Because Duchenne is a “rare disorder” (any disease or disorder with fewer than 200,000 cases in the United States), government funding is vital in our fight. Without governmental investment, a rare disorder like Duchenne will fall to the wayside. PPMD was instrumental in getting Congress to pass the Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act, the first legislation addressing muscular dystrophy. This monumental legislation was the catalyst for true investment into research, clinical trials, and care considerations for Duchenne. We continue to work with Congress to ensure that the bill is reauthorized.
EDUCATION Raising awareness is vital in our efforts to end Duchenne. In 2007, PPMD launched an outreach and awareness campaign designed to inspire people who aren’t affected by Duchenne to get involved, and rally those who are. The End Duchenne campaign turns our supporters into advocates and champions of our cause. Using new media online and off, we are giving our supporters opportunities to take action, invite friends and family to join the movement to end Duchenne, find new members of our community, post photos, and more.
AND AWARENESS
End Duchenne Campaign
THE PLEDGE TO
Every boy deserves to climb higher, walk farther, breathe deeper, and live longer.
I will support researchers until all boys with Duchenne can turn to an effective treatment.
I will advocate on behalf of families affected by Duchenne.
I will help connect the Duchenne community.
I will raise awareness about Duchenne.
I will join Parent Project Muscular Dystrophy in the fight to end Duchenne.
Sign online at EndDuchenne.org.
END DUCHENNE
EDUCATION In 2007, PPMD was honored by Oscar®-winning actress Olympia Dukakis’ appearance in a series of public service announcements entitled “Have You Seen This Boy?” Ms. Dukakis helps visitors recognize and understand the initial symptoms of Duchenne. The PSAs, released in 15-, 30-, and 60-second versions, are part of a concerted effort by PPMD and its partners to reach families and caregivers who may be unfamiliar with the symptoms, causes, and treatment of Duchenne. We’ve distributed them to television networks and major radio stations nationally and have already helped several previously undiagnosed families recognize symptoms and seek a diagnosis. Our goal is to reach young men as early as possible so that they can get the care necessary to have happy and fulfilling lives. You can view the PSA online on our media page.
Educational Outreach Program In a 2006 PPMD-sponsored national survey, less than 20% of respondents had heard of Duchenne muscular dystrophy. Of those that claimed to have heard of it, less than half could identify its symptoms. PPMD’s educational outreach program was created to address this lack of awareness. In 2007 PPMD greatly expanded its education outreach program. Funded through a grant from the Centers for Disease Control and Prevention, the program seeks to educate parents, educators, and healthcare professionals in recognizing symptoms of Duchenne, with the goal of early diagnosis and optimal treatment and care. At the suggestion of our Advisory Team, we selected the state of Mississippi to pilot the program (using the city of Jackson as our local hub). Engaging an active network of parent volunteers and local healthcare providers, the program has already been successful in reaching several families that had not yet received Duchenne diagnoses. We now have plans to expand these educational outreach efforts regionally and in several other locations nationally.
AND AWARENESS
Public Service Announcement: Have You Seen This Boy?
RUN FOR In response to the enormous success of our distance running sponsorship events, PPMD created the Run For Our Sons website, where runners, families, sponsors, and fans can come together to raise money for Duchenne research and treatment. Run for Our Sons also provides an important new way for members of the Duchenne community to come together and take action. Since 2005, more than 400 runners have joined Run For Our Sons teams, which participate in races across the country. They have raised more than $2.5 million for PPMD. With its combination of fundraising and training tips, racing opportunities and schedules, and personal blogs and stories, the Run For Our Sons website has flourished as more and more people discover the rewards of helping a truly worthy cause while having a great time and meeting wonderful people.
Running to End Duchenne Many people discover
Since that first half-marathon in 2006, Jen has
Run For Our Sons
followed up with fundraising races in 2007 and
while looking for running
2008. Along the way, she’s recruited sponsors
opportunities. Jen Alleva,
and friends to the cause. In fact,in just three
an accountant from
years, she and her friends have raised an
southern New Jersey,
estimated $30,000 for Duchenne research.
discovered running while looking for a way to help. After learning that her former classmate had a son with Duchenne, Jen joined her friend on a fundraising run for PPMD and was instantly hooked.
For Jen, the rewards are with her at every race. “Seeing families with their sons at the events warms my heart and makes me want to run even more.” And she’s not slowing down. Not only is she lining up more races with Run
Jen exemplifies the spirit behind the success
For Our Sons, she’s even gotten her whole
of Run For Our Sons. By her own admission,
family involved. At the 2007 Rocky Run, Jen
she’s not a professional athlete. “I don’t go
ran with her husband and three kids, as well as
very fast,” she laughs, “but I get there!” She
her old classmate and her son with Duchenne.
knows that every step she takes helps others.
OUR SONS
Run For Our Sons Website
COMMUNITY
PPMD is the only organization that hosts an annual conference each year so that our entire community of families, health professionals, and researchers can come together for discussion and support. Attendance at PPMD’s annual conference grows at a rate of approximately 20% each year, with participants learning, connecting, and sharing their experiences.
Connect Conference At PPMD’s Connect Conferences the remarkable, committed, and distinguished members of the Duchenne community come together to speak, listen, share, and learn. The conference serves as a terrific opportunity to see how much we have accomplished over the past year and to view the challenges ahead. We offer our deepest thanks to all who participate.
Conference Highlights Expert presentations, including • Drug development & regulatory practices • Latest care and treatment options • Advocacy • Research strategies Panel discussions, including • Communication and behavior • Global standards of care • Outcome measures for trials • Supplements and nutraceuticals
• Physical therapy • Technology Participatory activities, including • Practical demonstrations with qualified professionals: physical therapy techniques, cough assist, empowerment (advocating for your son), and more • The Rocky Run (a running celebration up the Philadelphia art museum steps made famous in the Rocky films) • Numerous get-acquainted meals and social events
More and more, we’re presenting material in a panel discussion format to allow for comparison and discussion among experts in the Duchenne field. Families benefit tremendously from these conversations, as well as from the opportunity of meeting other families dealing with similar issues.
Getting Together to End Duchenne Jennifer Garofalo is
presentations; he wants to know everything
a stay-at-home mom
about what’s happening at the cellular level.
in Middlesex, New
I’m more into the breakout sessions, where
Jersey with two sons.
I can meet other moms and dads and learn
Her oldest, Daniel,
practical ways of dealing with specific topics,
Jr., is 6 years old and
like behavioral issues. It’s incredibly helpful.”
has Duchenne. Jen and her husband went to their first PPMD Connect Conference in 2007 and can’t wait to go back.
Jen is looking forward to bringing her sons along when they’re a little older. “Seeing all the boys together, you realize that it’s one of the few times during the year that they get
“It means a lot, meeting all of the families,”
to feel “normal,” since they’re surrounded
she says. “And the all-inclusive nature of
by kids who are going through the same thing.”
the Connect Conference makes you feel as though you’re getting a better overall picture of the state of Duchenne research.” When asked what she and her husband value most about the conference, she says, “My husband loves the research
She pauses. “I think that’s true for the parents, too.”
HELP US
END DUCHENNE Get involved today. We offer numerous, simple ways to support the work of Parent Project Muscular Dystrophy: • Make a donation • Join S.T.I.R., our monthly giving plan • Invite others to make donations • Host an event • Join or support a Run For Our Sons team • Sign up at DuchenneConnect.org
• Advocate to your congressional representative • Teach others about Duchenne • Attend the conference • Attend our roundtable discussions • Visit ParentProjectMD.org to learn more
• Get active on the PPMD community site Next time you lift your fork, brush your teeth, hug a loved one, or even take a moment for that really satisfying yawn and stretch in the middle of the afternoon, think about a young man with Duchenne. The simplest movements—even just holding this book and flipping through the pages—can be complex and challenging activities.
Parent Project Muscular Dystrophy is leading the fight to end Duchenne. And your continued support is integral to our success. We must end Duchenne. And we will end Duchenne.
ParentProjectMD.org
LEADING THE FIGHT TO END DUCHENNE
Our Mission To improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, education, advocacy and compassion.
Parent Project Muscular Dystrophy 158 Linwood Plaza, Suite 220 Fort Lee, New Jersey 07024
T. 800.714.5437 • 201.944.9985 F. 201.944.9987
ParentProjectMD.org
created by Big Duck
PARENT MUSCULAR
PROJECT DYSTROPHY
For the Years Ended December 31, 2008 and 2007
Unrestricted Net Assets
2008
2007
$ 1,770,887
$ 2,368,742
Revenue and Other Support Grants and Contributions Program Revenue
60,209
72,108
Investment Gain
(1,615)
(1,126)
Interest and Dividend Income Fundraising Proceeds
3,136
5,057
2,087,505
2,231,574
3,920,122
4,676,355
3,633,050
3,894,249
(net of direct expenses of $681,383 and $431,685, respectively)
Total Revenue and Other Support Expenses Program Services Management and General Expenses
449,032
411,128
Fundraising Expenses
278,298
309,876
Total Expenses
4,360,380
4,615,253
Increase (Decrease) in Unrestricted Net Assets
(440,258)
61,102
540,141
479,039
FINANCIALS
STATEMENT OF ACTIVITIES
Net Assets Net Assets, End of Period
Copies of the full 2008 and 2007 audits are available upon request. See reverse side for Statement of Functional Expenses.
$
99,883
$
540,141
Parent Project Muscular Dystrophy ParentProjectMD.org
Net Assets, Beginning of Period
STATEMENT OF FUNCTIONAL EXPENSES For the Year Ended December 31, 2008 with Comparative Totals for 2007
Salaries Contract Labor Payroll Taxes
Research
Education
Advocacy
Mgmt. & Fundraising General
$ 242,217
259,952
536
72,958
3,500
–
–
–
2008 Total
2007 Total
62,654
638,317
586,012
–
3,500
6,900
24,059
20,511
41
7,642
6,223
58,476
50,384
Employee Benefits
–
–
–
18,588
–
18,588
16,112
Accounting Fees
–
1,715
–
57,538
15,000
74,253
75,839
–
320,252
48,614
–
–
368,866
335,619
39,178
1,725
–
40,345
11,388
92,636
23,463
Conferences and Meetings Legal Fees Bank Charges
9
–
–
18,633
59,339
77,981
28,444
Interest Expense
–
–
–
329
–
329
246
Fees and Permits
10,125
750
2,677
–
5,283
18,835
10,465
Insurance
–
7,908
–
60,673
–
68,581
64,016
Outside Services
–
47,000
–
1,800
2,300
51,100
57,355
Office and Computer Expenses
482
5,431
–
32,628
12,727
51,268
62,203
Technology
–
171,793
–
860
–
172,653
45,258
Rent
35,846
18,598
753
10,797
9,272
75,266
73,228
Postage and Shipping
–
27,918
–
3,588
7,176
38,682
44,730
DVDs and CDs
–
30,357
–
–
–
30,357
98,180
Printing and Publications
–
34,756
–
9,271
20,648
64,675
76,456
159,530
373,290
310,231
58,878
37,270
939,199
1,163,044
Telephone
–
24,157
349
3,451
6,902
34,859
25,519
Merchandise
–
–
–
–
12,324
12,324
9,567
139,781
59,912
8,484
37,271
5,822
251,270
328,760
Consulting Fees
Travel Meals and Entertainment
6,136
604
10,603
791
37,799
52,160
–
19,919
–
–
–
19,919
1,000
CDC Activity
–
23,757
–
–
–
23,757
Internet Access
–
–
–
–
–
–
674,392 1,455,837
372,289
445,853
– 89,310
Total Before Grants and Depreciation Grants Depreciation
275,119 3,223,490 3,324,270
812,816
314,441
–
–
–
1,127,257
1,281,849
–
3,179
96
3,179
3,179
9,633
8,832
–
–
–
–
–
–
302
$ 1,487,208 1,773,457
372,385
449,032
Loss on Disposal of Equipment Total Functional Expenses
278,298 4,360,380 4,615,253
Parent Project Muscular Dystrophy ParentProjectMD.org
19,665
Translation
John Killian, Chairman, Rockwall, TX Robert Nutt, Treasurer, Darien, IL Christine Piacentino, Secretary, Penfield, NY Neil Brandom, Newport Beach, CA David Drohan, Barrington, IL John Hiatt, Woodridge, IL Claudia Hirawat, South Plainfield, NJ Chuck Hurwitz, Lexington, MA Howard Kaplan*, Riverwoods, IL Brian Levin, Englewood, CO
BOARD AND STAFF
BOARD OF DIRECTORS
James Poysky, Houston, TX Donna Saccomano*, New Rochelle, NY Jeff Sobel, Chicago, IL Trent Spear, Oconomowoc, WI Donna Tomaselli, Highland Park, NJ Larry Weisman, Westport, CT * lifetime, non-voting board member
STAFF Kimberly Galberaith, Executive Vice President Will Nolan, Communications Director Ryan Fischer, Outreach Manager Sandra Goldman Janet Krakowski Sue Mahlock Stephanie Matthes Cecilia Matthes-Petersen Sandy Simpson
Parent Project Muscular Dystrophy ParentProjectMD.org
Pat Furlong, President, CEO