Learn about cystic fibrosis, a genetic disorder that affects the lungs, 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.
Research by dedicated scientists and clinicians from a wide range of disciplines is expanding our knowledge of cystic fibrosis, translating discoveries and insights into vital new treatments and clinical care practices for people living with CF.
Since Dr. Dorothy Andersen recognized cystic fibrosis as a disease in 1938, researchers have been trying to understand what causes CF and its complications and to find a cure. Families of people with CF realized early on that funding medical research would be essential to bring an end to the disease and created the National Cystic Fibrosis Research Foundation in 1955.
Although the name has changed, the mission of the Cystic Fibrosis Foundation remains the same. By pursuing a wide range of promising strategies, the Foundation has built a robust pipeline of approved and potential therapies that tackle CF from all angles, including drugs targeting the underlying cause of the disease. Nearly every drug recommended for CF was made possible because of CF Foundation support.
The Foundation supports the best research on both national and international fronts and from bench to bedside -- funding work on potential therapies in the laboratory and facilitating clinical trials to inform specialized care at CF Foundation-accredited care centers. This research includes:
Funding basic science research underpins our understanding of how CF affects the body. Scientists focus on explaining the mechanisms -- or the essential processes -- involved in causing the disease and its complications, such as CF-related diabetes.
The Foundation supports leading scientists at universities around the world to study everything from the structure of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to the challenges associated with editing the CFTR gene. In addition, the Foundation supports research and development programs -- or research hubs -- that focus on critical scientific problems and train the next generation of CF scientists. Every year, many of the labs that receive grants present their research findings at the annual CF Foundation Research Conference.
The CF Foundation operates its own research laboratory dedicated exclusively to advancing CF therapies. The Cystic Fibrosis Foundation Therapeutics Lab capitalizes on emerging scientific discoveries related to CFTR modulators, nonsense mutations, gene editing, and stem cell research. It develops drug-screening tools and technologies identified by scientists and makes them available to other researchers and companies. In addition, the lab team trains external researchers all over the world in the specialized techniques needed to develop CF therapies.
Sound investments by the Foundation in cutting-edge science have helped build an extensive base of knowledge about the disease. Some of these ideas have led to innovative new therapies in the Foundation's Drug Development Pipeline that have made a significant difference in the health and quality of life of tens of thousands of people who have CF.
Because pharmaceutical companies have historically been reluctant to make major investments in developing drugs for rare diseases like CF, the Foundation has provided funding to encourage biotech and pharmaceutical companies to focus on CF and help advance potential therapies that would be unlikely to move beyond the lab and into clinical trials. The Therapeutics Development Program provides funding during the early phase of drug discovery and development, when the risk for failure is greatest and companies are the most hesitant to invest. These efforts have resulted in CF drugs, such as dornase alfa (Pulmozyme®), inhaled tobramycin (TOBI®), and ivacaftor (Kalydeco®).
The Foundation also funds clinical researchers who analyze the most promising research findings to develop potential new treatments. For example, the Foundation is supporting drug discovery efforts to screen compounds to see if they can “read through” the premature stop signals caused by nonsense mutations. If so, these compounds will be developed further into potential drugs and then tested in animals to help determine safety. This type of pre-clinical work -- before a drug can be tested in people -- is essential in drug development.
If the animal studies are successful, clinical researchers then set up clinical trials to test these potential therapies in people. To speed up the testing of these drugs, the Foundation provides funding to the Therapeutics Development Network (TDN), a network of 89 Foundation-accredited care centers with specialists in CF clinical research who recruit study participants and conduct clinical trials. (Our Drug Development Pipeline shows drugs that the Foundation has either helped financially or assisted through the TDN.)
In addition, the Foundation has been working with the U.S. Food and Drug Administration (FDA) to develop strategies to test new therapies even when only a small number of people with CF are affected. These strategies are especially needed to help people with CF who have very rare mutations.
Real-world research takes into account the realities of daily life and human behavior and how they can affect the way treatments work and the way care is provided. At the CF Foundation, we conduct different types of real-world research, including:
Observational studies conducted with data from the CF Foundation's Patient Registry help us understand CF by showing trends in survival and complications among people with CF who volunteer their information and are seen at CF Foundation-accredited care centers. The studies also allow us to track the effectiveness of therapies. Unlike interventional studies where potential treatments are tested, observational studies do not involve any changes to a treatment plan and are designed for observing individuals in their normal day-to-day settings, while measuring the effects or outcomes of those treatments.
Another example of real-world research supported by the Foundation is the post-approval study. After a drug has been approved by the FDA, researchers will continue to monitor the drug for safety and effectiveness in what is known as a Phase 4 study. These studies look at the long-term effects of the therapy to further refine the safety, efficacy, or usage of the treatment as people use it in their day-to-day or “real-world” settings.
The Foundation also funds comparative effectiveness research on FDA-approved therapies to collect data on which ones help people with CF the most. These research findings are used to inform best practices at CF care centers nationwide. For example, in the STOP 2 trial, researchers are trying to determine the ideal amount of time to administer IV antibiotics to treat pulmonary exacerbations.
Behavioral studies are designed to identify how factors, such as self-management, mental health, and social support, are associated with health outcomes. Understanding these factors help us develop ways to improve therapies and how they are used. Researchers can observe how people take their medications or follow a daily care plan to see how it affects their health. Behavioral studies can be observational, where there is no change in how or what people do with regard to their treatment plan, or interventional, where researchers monitor a change in behavior to determine if it has an impact on the health of people with CF.
At the CF Foundation, the Success with Therapies Research Consortium (STRC) is conducting behavioral studies to identify new tools and practices to help care teams partner with people with CF to more effectively manage their disease.
Dr. Kristin Riekert, STRC co-chair, discusses how the STRC is working to develop tools to help people with CF manage their daily care more effectively and when they might be available for people with CF.
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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