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 people living with cystic fibrosis and their families face complicated issues related to getting the care they need. Cystic Fibrosis Foundation Compass makes sure that no one has to do it alone.
CF Foundation Compass is a service that helps people with CF and their families with navigating insurance options, connecting to legal information and experts, finding available financial resources, and tackling other life issues.
CF care team members are paramount in providing highly specialized care to people living with CF. CF Foundation Compass can help by serving as a strategic ally for care teams, so team members can focus on their patients’ care.
CF Foundation Compass can help you navigate insurance, financial, legal, and other issues you are facing. Use this online form to start your conversation with a Compass case manager today.
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.
Today, the U.S. Food and Drug Administration approved the use of ivacaftor (Kalydeco®) for children with cystic fibrosis ages 1 to 2 who have at least one mutation that is responsive to ivacaftor.
Published on August 15, 2018
Today, the U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) for children with cystic fibrosis ages 1 to 2 who have at least one mutation that is responsive to ivacaftor. This includes the 38 mutations already approved for children ages 2 and older.
Ivacaftor is a type of cystic fibrosis transmembrane conductance regulator (CFTR) modulator called a potentiator that increases activity of the CFTR protein at the cell surface and has been shown in studies to improve lung function.
“Today's approval means we can work to prevent the onset of lung disease in eligible children as early as their first birthday,” said Michael P. Boyle, M.D., senior vice president of therapeutic development for the Cystic Fibrosis Foundation. “In the next few years, we hope these life-changing treatments that target the underlying cause of the disease are expanded to more than 90 percent of people with CF.”
This news follows the FDA's approval on Aug. 7 of the use of lumacaftor/ivacaftor (Orkambi®) for children with CF ages 2 to 5 who have two copies of the most common CF gene mutation, F508del. That expansion allowed 1,300 additional children in the U.S. to become eligible to receive the drug.
The approval of ivacaftor was informed by a positive study in children ages 1 to 2. Ivacaftor was generally well tolerated in the 24-week study, and there were no new safety concerns. Although safety was the primary focus of the study, it also found that the children's sweat chloride was significantly reduced to within the normal range during the trial.
In addition, the children in the study had substantial improvements in their levels of fecal elastase (a type of digestive enzyme). Fecal elastase levels are often used as a measurement of how well the pancreas is working. These data suggest that early treatment with ivacaftor may help preserve pancreatic function. A study of ivacaftor in infants ages 1 and younger is ongoing.
Ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.
The Foundation also supports research aimed at discovering new treatments for people with nonsense and rare mutations that do not produce the CFTR protein, an estimated 5 to 7 percent of people with CF. These individuals will be unable to benefit solely from CFTR modulators. In 2016, the Foundation launched the Nonsense and Rare Mutations Research and Therapeutics Initiative to help advance research by academic institutions and pharmaceutical companies focused exclusively on the creation of therapies to “restore” CFTR production for these individuals.
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