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 Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
Published on January 31, 2018
The cystic fibrosis transmembrane conductance regulator (CFTR) modulator candidates VX-659 and VX-445 will advance into Phase 3 clinical trials as part of two different triple combination therapies. The decision to proceed with these two modulators was based on data from recent Phase 2 clinical trials that showed these candidates performing better than previous contenders.
The Phase 2 studies for VX-659 and VX-445, for which early results were announced today, examined the effectiveness of each compound in combination with tezacaftor and ivacaftor in individuals with one F508del CFTR mutation and one minimal function mutation. Participants in these clinical trials who took the highest dosage of VX-659 and VX-445 as part of a triple combination saw improvements in their FEV1 by an average of 13.3 percent and 13.8 percent respectively after four weeks of treatment. Both groups also experienced significant improvements in their sweat chloride levels and their quality of life measurements.
“The research released today underscores the potential of next-generation combination therapies to be significantly more effective than current modulators,” said Michael Boyle, M.D., senior vice president for therapeutics development for the Cystic Fibrosis Foundation. “Importantly, the ability of these potential drugs to treat individuals with a single F508del mutation means that more people than ever before could benefit. This is very exciting news for our community, and we are grateful to those who participated in the clinical trials that made today's announcement possible.”
Vertex Pharmaceuticals Inc. anticipates that the first of the Phase 3 clinical trials for VX-659 in combination with tezacaftor and ivacaftor will begin enrollment in the first half of 2018. The studies will be conducted in people with one F508del mutation and one minimal function mutation, as well as in people with two F508del mutations. Vertex also plans to begin a Phase 3 clinical trial for VX-445 in combination with tezacaftor and the compound VX-561 as a once-daily therapy in people with one F508del and one minimal function mutation, and in people with two F508del mutations, in mid-2018.
“Today's announcement highlights the tremendous potential of the triple combination therapy to address the underlying cause of the disease in more people with CF than ever before,” said Preston W. Campbell, III, M.D., president and CEO of the Cystic Fibrosis Foundation. “We are extremely excited by the prospect of reaching over 90 percent of the CF population with these life-saving modulators and are committed to ensuring that all people with CF will one day have disease-modifying treatments.”
The Cystic Fibrosis Foundation provided $150 million in research funding to Vertex to support its pursuit of treatments that address the underlying defect in CF, including $75 million to develop next-generation candidates currently in clinical development.
For additional information regarding the results of these clinical trials, including the safety and efficacy outcomes, please see the Vertex press release.
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