LEADING THE FIGHT

TO END DUCHENNE

RUN FOR OUR SONSP

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ANNUAL CONFERENCE

CORPORATE SPONSORSHIPS

PTC-124

REGIONAL ROUNDTABLE

COMMUNITY

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PROGRESS REPORT

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MD-CARE ACT

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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

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SDear 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 long-

term 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

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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 long-

term therapies;

• To leverage $200 million into re-

search and centers and $22 million

into Duchenne-specific programs

at the Centers for Disease Control

and Prevention;

• 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.

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.

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• 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

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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 long-

term 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

RECENT ACHIEVEMENTS

• Launched pilot outreach program in

Mississippi in partnership with the Centers

for Disease Control and Prevention to

º Raise awareness and reach out to families

who may not yet have received a Duchenne

diagnosis, and those who may be receiving

inadequate care

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

to both DuchenneConnect.org, and

ParentProjectMD.org, while broadly raising

awareness for Duchenne at no cost

• Continued to expand Regional Roundtables,

providing up-to-date information on Duchenne

research and care, including the first-ever online

“Virtual Roundtable,” which reaches a broader

audience that simply logs in online to listen to

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

RECENT ACHIEVEMENTS

• 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.”

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Gresearch initiatiVe FundingProject 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.

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inductiOn OF giOVanna spinella Creating a standard

of care for Duchenne

patients has been a

long-standing need

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.

Dr. Spinella is a pediatric neurologist with

16 years of experience at the National

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

program director for that office. Dr. Spinella

is a resident of Falls Church, Virginia, and

until recently served as staff neurologist at

Walter Reed Army Medical Center.

“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,

a standard approach is needed to get

accurate data.”

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.

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cOnnecting tO end duchenne John and Kristin Hiatt

knew something was

wrong with their son

Liam. The symptoms

led them to suspect

Duchenne, but doctors

in Chicago hospitals

told them it was

simple motor delay. Unconvinced, they hit the

Internet and discovered PPMD’s website.

Upon recommendations from Pat Furlong

(and other parents) they contacted Dr. Brenda

Wong, Doctor of Neurology at Cincinnati

Children’s Hospital Medical Center, and

made the four-hour drive from the Chicago

area for testing.

“If we could clone Dr. Wong and put her

(and CCH’s standards of care) in the Chicago

hospital system, it would save us a very long

commute,” says John, “but it’s well worth the

trip. Their approach is comprehensive and

proactive. For example, we now have a

cough assist machine to help keep Liam’s

lungs clear. If we’d followed our Chicago

doctors’ advice, we would have waited until

he developed pneumonia.”

The Hiatts are also registered members

of DuchenneConnect. “Other MD databases

confuse potential registrants with issues

of eligibility and present great difficulty in

getting quick answers to questions.

DuchenneConnect is set up clearly and

is easier to use and understand.”

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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

Act, the first legislation for muscular

dystrophy. It is also the only condition-

specific legislation to become law in

recent years

• Since passage of MD-Care Act, more

than $200 million has been invested in

muscular dystrophy research with more

than $45 million going to Duchenne-

specific research

• A total of six Wellstone Centers of

Excellence have been established

because of MD-Care Act

• PPMD’s annual Advocacy Conference

unites hundreds of families from across

the United States and is the only

organized Duchenne advocacy event

• PPMD’s advocates visit hundreds of

congressional offices annually urging

an increase in funding at the National

Institutes of Health and the Centers for

Disease Control and Prevention

• Pat Furlong is a board member of the

MDCC (Muscular Dystrophy Coordinating

Committee), and a committee member

on the Collaboration in Education and

Test Translation Program

• Pat Furlong also serves on the data

safety monitoring board for both the

Rare Diseases Clinical Research Network

and Cooperative International Neuro-

muscular Research Group

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.

end duchenne caMpaign 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.

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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.

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public serVice annOunceMent: haVe yOu seen this bOy? 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.

run FOr Our sOns Website 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

Run For Our Sons

while looking for running

opportunities. Jen Alleva,

an accountant from

southern New Jersey,

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.

Jen exemplifies the spirit behind the success

of Run For Our Sons. By her own admission,

she’s not a professional athlete. “I don’t go

very fast,” she laughs, “but I get there!” She

knows that every step she takes helps others.

Since that first half-marathon in 2006, Jen has

followed up with fundraising races in 2007 and

2008. Along the way, she’s recruited sponsors

and friends to the cause. In fact,in just three

years, she and her friends have raised an

estimated $30,000 for Duchenne research.

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

For Our Sons, she’s even gotten her whole

family involved. At the 2007 Rocky Run, Jen

ran with her husband and three kids, as well as

her old classmate and her son with Duchenne.

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RUN FOR

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.

COMMUNITY

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

getting tOgether tO end duchenne

More and more, we’re presenting material in a panel discussion format to allow for com-

parison 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.

Jennifer Garofalo is

a stay-at-home mom

in Middlesex, New

Jersey with two sons.

Her oldest, Daniel,

Jr., is 6 years old and

has Duchenne. Jen

and her husband went

to their first PPMD Connect Conference in

2007 and can’t wait to go back.

“It means a lot, meeting all of the families,”

she says. “And the all-inclusive nature of

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

presentations; he wants to know everything

about what’s happening at the cellular level.

I’m more into the breakout sessions, where

I can meet other moms and dads and learn

practical ways of dealing with specific topics,

like behavioral issues. It’s incredibly helpful.”

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

to feel “normal,” since they’re surrounded

by kids who are going through the same thing.”

She pauses. “I think that’s true for the

parents, too.”

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

• Get active on the PPMD community site

• Advocate to your congressional representative

• Teach others about Duchenne

• Attend the conference

• Attend our roundtable discussions

• Visit ParentProjectMD.org to learn more

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.

END DUCHENNEH

ELP

US

ParentProjectMD.org

LEADING THE FIGHT TO

END DUCHENNE

ParentProjectMD.org

Our MissiOnTo 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 Dystrophy158 Linwood Plaza, Suite 220Fort Lee, New Jersey 07024

T.800.714.5437•201.944.9985F.201.944.9987

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STATEMENT OF ACTIVITIES For the Years Ended December 31, 2008 and 2007

Unrestricted Net Assets 2008 2007

Revenue and Other Support

Grants and Contributions $ 1,770,887 $ 2,368,742

Program Revenue 60,209 72,108

Investment Gain (1,615) (1,126)

Interest and Dividend Income 3,136 5,057

Fundraising Proceeds 2,087,505 2,231,574

(net of direct expenses of $681,383 and $431,685, respectively)

Total Revenue and Other Support

3,920,122

4,676,355

Expenses

Program Services

3,633,050

3,894,249

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

Net Assets Net Assets, Beginning of Period

540,141

479,039

Net Assets, End of Period

$ 99,883

$ 540,141

Copies of the full 2008 and 2007 audits are available upon request. See reverse side for Statement of Functional Expenses.

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STATEMENT OF FUNCTIONAL EXPENSES For the Year Ended December 31, 2008 with Comparative Totals for 2007

Research Education Advocacy Mgmt. & Fundraising 2008 2007 General Total Total

Salaries $ 242,217 259,952 536 72,958 62,654 638,317 586,012

Contract Labor 3,500 – – – – 3,500 6,900

Payroll Taxes 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

Conferences

and Meetings – 320,252 48,614 – – 368,866 335,619

Legal Fees 39,178 1,725 – 40,345 11,388 92,636 23,463

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

Consulting Fees 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

Travel 139,781 59,912 8,484 37,271 5,822 251,270 328,760

Meals and

Entertainment 19,665 6,136 604 10,603 791 37,799 52,160

Translation – 19,919 – – – 19,919 1,000

CDC Activity – 23,757 – – – 23,757 –

Internet Access – – – – – – 89,310

Total Before Grants

and Depreciation 674,392 1,455,837 372,289 445,853 275,119 3,223,490 3,324,270

Grants 812,816 314,441 – – – 1,127,257 1,281,849

Depreciation – 3,179 96 3,179 3,179 9,633 8,832

Loss on Disposal

of Equipment – – – – – – 302

Total Functional

Expenses $ 1,487,208 1,773,457 372,385 449,032 278,298 4,360,380 4,615,253

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FFBOARD OF DIRECTORS 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

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

Pat Furlong, President, CEO

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

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