Learn about cystic fibrosis, a genetic lung disorder that affects the pancreas and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
Our goal is to educate policy makers about the needs of people with cystic fibrosis so that they make smart decisions about CF-related research, treatment, and access to care.
We recognize the value of tapping into the expertise that only people with CF and their families have. We invite you to share insights to help improve and develop programs and services that support the daily lives of people with CF.
Our mission is to find a cure for cystic fibrosis and improve the quality of life for those living with the disease. We can't do it alone. Help us add tomorrows by giving today.
In addition to working for a cure, the CF Foundation supports programs and policies to improve the lives of people with CF. Help us by raising awareness of CF, participating in a fundraising event, or volunteering with your local chapter.
In 2016, the Cystic Fibrosis Foundation spent more than $28 million on laboratory research and clinical trials for new and more effective drugs to restore the function of the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein.
For nearly 20 years, the Foundation has pursued a long-term strategy to develop effective treatments that target the underlying defect in cystic fibrosis for all types of mutations. Currently, there is an unprecedented number of new and potentially more effective CFTR modulators being tested in clinical trials.
The current generation of U.S. Food and Drug Administration (FDA)-approved CFTR modulators, ivacaftor (Kalydeco®) and lumacaftor/ivacaftor (Orkambi®), will eventually benefit more than 60 percent of people with CF, and trials are underway to determine the effectiveness and safety of these drugs in children as young as 6 months old. Gaining access to these drugs early in life is an important step, as new research indicates that taking modulators before the disease has progressed could potentially preserve long-term lung function.
At the beginning of 2017, two Phase 3 clinical trials for the first-generation modulator tezacaftor (VX-661), in combination with ivacaftor, showed promising results. If approved, tezacaftor/ivacaftor will offer a new option for first-generation modulators with potentially fewer side effects than lumacaftor/ivacaftor (Orkambi). Tezacaftor is also positioned to play a critical role in next-generation triple-combination therapies.
Currently, there are a number of new and potentially more effective CFTR modulators (drugs that treat the underlying defect that causes the disease) being tested in clinical trials. The goal of these next-generation therapies, which may include a three-drug combination, is to develop treatments that are more effective and benefit more people, including individuals with only one copy of the F508del mutation.
Individually, each of the three compounds in the triple-combination therapies addresses a different aspect of the underlying defect. When used in combination, the results are magnified. Already, several next-generation modulators in development have the potential to be significantly more effective than current FDA-approved modulators and to also benefit more people with CF than ever before.
In July 2017, early stage clinical trial results for three of these triple-combination therapies showed striking improvements in lung function in participants with only one copy of the F508del mutation. The therapies also demonstrated significant improvement in lung function in participants with two copies of the F508del mutation. Based on these results, the CF Foundation anticipates that within the next decade over 90 percent of people with CF could benefit from modulators either as effective, or more effective, than ivacaftor.
Next-generation Phase 3 clinical trials should begin in mid-2018. Other companies also are planning Phase 1 trials for additional modulators next year.
Research is underway to categorize (or group) mutations by how they respond to different modulators. By testing modulators on cells with specific CFTR mutations, scientists can identify new possible treatments for people with mutations that are so rare as to make clinical trials impractical. This technique, called “theratyping,” could eventually enable doctors to tailor, or “personalize,” treatments based on how the drugs affect individual mutations in lab tests.
The Foundation is working with scientists, clinicians, and regulators to devise ways to use laboratory theratype data to help expand approval of current modulators on the market to as many people with CF as possible.
Testing is already underway at three Foundation-funded labs to see if 650 rare CFTR mutations can be corrected by a modulator. If any of the tests show positive results, the Foundation will provide the information to the relevant drug company in order to assist the FDA approval process.
Theratyping has already proven successful. In early 2017, the FDA relied on a combination of theratyping results, clinical data, and the drug's established safety record to expand the use of ivacaftor (Kalydeco®) to people ages 2 or older who have at least one of 23 CFTR mutations.
Reference to any specific product, process, or service does not necessarily constitute or imply its endorsement, recommendation, or favoring by the Cystic Fibrosis Foundation. The appearance of external hyperlinks does not constitute endorsement by the Cystic Fibrosis Foundation of the linked websites, or information, products, or services contained therein.
Information contained on this site does not cover all possible uses, actions, precautions, side effects, or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
FDA-approved drug information is available at dailymed.nlm.nih.gov/dailymed.
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