Imagine a Cure for Cystic Fibrosis PDF 1 MB

FOR CYSTIC F IBROSIS

Imagine a

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SUTTON, AGE 2


Each year, parents across the country will receive the same sobering diagnosis from their doctor:

Your child has cystic fibrosis— a rare, genetic disease that progressively limits the ability to breathe and tragically shortens lives.

This moment marks the beginning of a long and challenging journey for CF families.


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leading the wayTHE CYSTIC FIBROSIS FOUNDATION,

The Cystic Fibrosis Foundation is here to help

those living with this devastating disease. In

people with CF, a defective gene causes a thick,

buildup of mucus in the lungs, pancreas and

other organs. In the lungs, the mucus clogs the

airways and traps bacteria leading to infections,

extensive lung damage and eventually,

respiratory failure. Sixty years ago, most children

did not live long enough to attend elementary

school, but thanks to Foundation-based research

and care, the median survival age of people

with CF is now about 47.

The CF Foundation, based in Bethesda, Md.,

with over 70 chapters across the country, was

founded in 1955 by parents desperate to save

their children’s lives. Their relentless and

impassioned determination to prolong life has

resulted in tremendous strides over the past 60

years in accelerating innovative research and

drug development, as well as advancing care and

advocacy. Virtually every approved cystic fibrosis

drug therapy available now was made possible

because of the Foundation and its supporters.

Still, we believe no one should have to die at a young age. We will not rest until we have a cure for all people living with CF.


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Sixty years ago, most children did not live long enough to

attend elementary school, but thanks to Foundation-based

research and care, the median survival age of people with

CF is now about 47.

BREE, AGE 5

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EVERY DAY IS A CHALLENGE

Cystic fibrosis is a complex disease and the

types and severity of symptoms can differ

widely from person to person. While we have

achieved enormous gains in quality of life and life

expectancy, living with cystic fibrosis is not easy.

Morning and bedtime routines are typically filled

with time-consuming treatments to keep lungs

healthy and help break up the sticky mucus that

impairs breathing and invites life-threatening

infections. For many with CF, frequent

hospitalizations mean missed school and work

days and major interruptions to “normal” life.

Thankfully, because of the Foundation’s efforts

over the past six decades, many of these

individuals are living long enough to realize

their dreams of attending college, pursuing

careers, getting married and having children.

Yet, there is currently no cure for CF.

we are accelerating…

MEAGHAN, AGE 3

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Groundbreaking Research Drugs that treat the genetic cause of CF are

rapidly becoming available to more individuals

as a result of the Foundation’s efforts. And, we

are exploring exciting new technologies such as

gene editing, RNA therapy and stem cell biology

that could pave the way toward a one-time cure

for all people with CF.

High-quality Care We help advance excellent care for those living

with CF through our network of more than 120

accredited care centers and 55 affiliate programs.

These care centers, located in hospitals and

medical facilities across the country, deliver

specialized care to those with CF.

Effective AdvocacyWe are a tenacious advocate for people with

CF and fight for programs, agencies and

policies that help advance research and drug

development, improve access to specialized,

quality CF care and raise awareness about

the disease.

Strong Community Partnerships We listen to CF families to ensure that our

programs remain meaningful. Community

Voice — composed of nearly 800 people across

the country affected by CF— regularly conveys

the hopes and aspirations of adults living with

this disease so we can continually offer support

that enhances lives.

…the search for a cureEVERY BREAKTHROUGH BRINGS HOPE

While the Cystic Fibrosis Foundation is blazing new trails in the search for a cure to CF, we are also working to make a real difference in the daily lives of people living with this disease.

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ABOUT CF

A PROMISING PIPELINE IN DRUG DEVELOPMENT

The CF Foundation continues to make tremendous progress in pursuing its bold research agenda to tackle CF from all angles. Drugs that treat the underlying

genetic cause of the disease are becoming available to more individuals, and promising treatments to manage the symptoms of CF are advancing in the pipeline.

Available to Patients

Phase 3 Definitive Trial

Phase 2 Human Safety & Efficacy Trial

Phase 1 Human Safety Trial

Pre-Clinical (Initial Testing in Laboratory)

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RESTORE CFTR FUNCTIONJuly 2016 MUCOCILIARY CLEARANCE

ANTI- INFLAMMATORY ANTI-INFECTIVE NUTRITIONAL-GI

MORE THAN

30,000 people

are living with cystic fibrosis (more than 70,000 worldwide).

APPROXIMATELY

1,000 new casesof CF are diagnosed

each year.

MORE THAN

10 million Americans

are symptomless carriers of the defective CF gene.

MORE THAN

halfof the CF population

is age 18 or older.

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KIRAN, AGE 1

“ I dream of the day that the CF Foundation puts an end to this disease. I want to see my loving son have a long and healthy life, like all children deserve.”

KIRAN’S DAD


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YOU CAN MAKE A DIFFERENCE

Over the past 25 years alone, it has taken almost

$3 billion for the Foundation and Cystic Fibrosis

Foundation Therapeutics — our nonprofit drug

discovery and development affiliate — to achieve

the results we have today, including dramatic

improvement in median life expectancy and

two disease-modifying therapies.

We will continue to invest heavily in the science

that directly supports our mission. What we

achieve going forward will not only help us find

a cure for CF, but may also lead to advances in

research for people with other diseases. As we

embark on the next leg of our journey, we invite

you to join us.

GIVE TODAY

You are critical to our progress. However big

or small your contribution is, every gift makes

an impact. There are many options for you to

consider. Whether you want to make a quick,

one-time donation, become a monthly donor

or make a legacy gift in your will — we value

your donation.

You can also volunteer your time, become a

CF advocate or participate in one of our many

events across the country.

Visit www.cff.org/give or call 1.800.FIGHT.CF.

the possibilitiesimagine

We are at a pivotal moment in the history of cystic fibrosis, with promising therapies on the horizon. While people with CF are living longer than in the past, we still lose precious young lives every day.

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PAGE 12 – BACK COVERSomeone said that the Instagram icon is old – that there is a newer one – if that’s true, would you make sure we have whatever is most current today?

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RYAN, AGE 4


4550 MONTGOMERY AVE,

SUITE 1100 N

BETHESDA, MD 20814

1.800.FIGHT.CF

W W W.CFF.ORG

twitter.com/CF_Foundation

facebook.com/CysticFibrosisFoundation

instagram.com/CF_Foundation

youtube.com/CysticFibrosisUSA

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the

disease the opportunity to lead full, productive lives by funding research and drug development, promoting

individualized treatment and ensuring access to high-quality, specialized care.


Documents

Imagine a Cure for Cystic Fibrosis PDF 1 MB