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.
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
Published on June 9, 2021
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children with cystic fibrosis ages 6 through 11 who have at least one copy of the F508del mutation. Trikafta was also approved for children ages 6 through 11 who have certain mutations in the CFTR gene that are responsive based on lab data. With this approval, approximately 1,500 children will be eligible for a CFTR modulator for the first time.
“This approval is wonderful news for our community, especially for the families of those young children with CF who can now benefit from a modulator for the first time,” said Michael P. Boyle, MD, president and CEO of the Cystic Fibrosis Foundation. “We have seen the transformative effect that Trikafta can have on older children and adults, and we are excited by the prospect that this medicine may slow or prevent the progression of the disease at an early age and stave off the most common complications of CF before they start.”
Highly effective CFTR modulators, such as Trikafta, are known to have a transformative effect on the health and well-being of many people with CF. For example, studies have shown that some young children taking Kalydeco® (ivacaftor) have had their pancreatic function restored.1 But these therapies do not reverse established lung damage. This is one of the reasons it is important to start on CFTR modulators at a young age. Research suggests that beginning treatment with a modulator early could help slow or even prevent the irreversible progression of cystic fibrosis, dramatically altering the course of disease over time.
The FDA approval was based on data from a 24-week Phase 3 study, which was evaluating the safety and efficacy of Trikafta in children ages 6 to 11 with at least one F508del mutation. The study showed that the drug was generally well tolerated, and the safety data were consistent with those observed in previous studies. The FDA also approved Trikafta for children who have one of 177 other mutations that have been studied in the laboratory. The same laboratory data helped form the basis for approval of Trikafta for those ages 12 and older in December 2020.
For additional information, please see the Vertex press release.
Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease, including those who won't benefit from modulators such as Trikafta. The Cystic Fibrosis Foundation's $500 million Path to a Cure is focused on developing new treatments for the underlying cause of the disease and, one day, a cure. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF.
1Nichols AL, Davies JC, Jones D, Carr SB. Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor. Paediatr Respir Rev. 2020 Sep;35:99-102. doi: 10.1016/j.prrv.2020.04.003. Epub 2020 Apr 14. PMID: 32386958.
Munce D, Lim M, Akong K. Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor. Pediatr Pulmonol. 2020 Dec;55(12):3381-3383. doi: 10.1002/ppul.25065. Epub 2020 Oct 22. PMID: 32910556.
Aoyama BC, Mogayzel PJ. Ivacaftor for the treatment of cystic fibrosis in children under six years of age. Expert Rev Respir Med. 2020 Jun;14(6):547-557. doi: 10.1080/17476348.2020.1741352. Epub 2020 Mar 17. PMID: 32154747.
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