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.
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.
The symptoms of cystic fibrosis are caused by a defective protein, known as the cystic fibrosis transmembrane conductance regulator (CFTR). Researchers are investigating potential therapies to restore proper function to the CFTR protein or correct its production process so that a normal protein is made.
A properly functioning cystic fibrosis transmembrane conductance regulator (CFTR) protein forms a channel that allows chloride (a component of salt) to move in and out of cells lining the lungs and other organs. The CFTR protein is shaped like a tube that goes through the membrane surrounding the cell, like a straw goes through the plastic top on a cup.
Different CF mutations cause the protein to malfunction in different ways. New and potential therapies target these specific problems, sometimes by using more than one drug, to restore CFTR function and help improve key symptoms of CF.
Ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), and tezacaftor/ivacaftor (Symdeko®) make up the first generation of CFTR modulators, which seek to restore function to the CFTR protein.
Watch this animation to see how CFTR modulators known as correctors and potentiators work to make the CFTR protein functional.
Learn about the science behind the cystic fibrosis drugs that are in development for people with just one copy of the F508del mutation.
There are several next-generation modulators currently in clinical trials, including two triple-combination drugs made up of ivacaftor, tezacaftor, and either VX-445 or VX-659. Results from both Phase 3 clinical trials for these triple-combination drugs are expected by early 2019. Previous clinical trial results have indicated that these triple-combination modulators will likely be more effective than current modulators and could potentially treat a much larger population of people with CF.
The goal is to find a treatment that would help all people with CF. The first-generation modulators could benefit 61 percent of people with CF in the U.S. if all age groups are approved. Next-generation triple-combination therapies could help people with just one copy of the F508del mutation, potentially increasing the number of people who could benefit from modulators to more than 90 percent.
In this video, George M. Solomon, M.D., assistant professor of medicine and director of the adult CF program at the University of Alabama at Birmingham, explains what researchers are doing to try to develop CFTR modulators that can treat a wider variety of mutations.
It is estimated that 5 to 7 percent of people with CF will be unable to benefit solely from CFTR modulators and will require different therapies to fix the underlying cause of their disease. We are funding research aimed at discovering new treatments for these individuals with nonsense and rare mutations that do not produce CFTR protein.
Preclinical work is being done to screen for compounds that could enable the protein-making machinery of the cell to override premature stop signals caused by nonsense mutations so that a full-length protein can be made. These compounds would be known as readthrough agents because they could “read through” the premature stop signals.
Watch this animation to see how nonsense mutations might be corrected to make normal CFTR protein.
The multiple approaches reflected in the Drug Development Pipeline include increasing the amount of CFTR protein that reaches the cell surface, stabilizing the protein so that it functions better, and repairing or replacing the CFTR-encoded messenger ribonucleic acid (mRNA) that is used as a template to create the CFTR protein.
The latter treatment is known as RNA therapy, and it could potentially benefit everyone with CF regardless of their mutations. For RNA therapy, the Foundation is pursuing two different approaches in parallel. The first approach would replace the defective mRNA by delivering normal mRNA to cells. The second approach is to repair the defective mRNA responsible for producing the CFTR protein. Both strategies would enable full-length, functional CFTR protein to be made.
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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