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.
Two Phase 3 clinical trials of tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) showed positive results, Vertex Pharmaceuticals announced today.
Published on March 28, 2017
Results released from the first of the large Phase 3 clinical trials of the drug combination demonstrated that people with two copies of the F508del mutation ages 12 and older who received the treatment had significant improvements in lung function -- 4 percentage points compared to placebo -- and in other key measures of the disease, including exacerbations (sudden worsening of symptoms) and quality of life.
Tezacaftor/ivacaftor was well tolerated, with an overall incidence of side effects similar to that seen in the placebo treated group. Additionally, respiratory complications were similar between placebo and treatment groups.
The 24-week study was conducted at over 90 clinical trial sites in North America and Europe. In total, more than 500 people with two copies of the F508del mutation, ages 12 and older participated in the study.
The second of the Phase 3 clinical trials tested the safety and effectiveness of tezacaftor in combination with ivacaftor for people with one F508del mutation and a second CFTR mutation that results in residual function. (Residual function mutations result in some CFTR function being preserved because CFTR protein is present at the cell surface but is either not fully active or not present in sufficient amounts).
The eight-week study showed that, compared with those on placebo, participants with one F508del and one residual function mutation who took the combination treatment improved lung function by 6.8 percentage points. The study also tested ivacaftor without tezacaftor. Participants who only received ivacaftor had a 4.7 percent improvement in lung function compared to placebo.
The residual function mutations eligible for inclusion in the residual function trial were chosen using pre-clinical analysis that identified them as likely to respond to ivacaftor. The list of residual function mutations eligible for inclusion in the trial can be found here. As there are no drugs approved by the U.S. Food and Drug Administration (FDA) to treat the underlying defect of the disease for people with these residual mutations, the more than five percent of people with CF who have these mutations could eventually benefit from modulator therapy.
"These positive results represent a significant step forward in our effort to bring new and more effective treatments targeting the underlying cause of the disease to all people with CF," said Preston W. Campbell, M.D., president and CEO of the CF Foundation. "We have a tremendous amount of momentum right now, and we are determined to build on it to create effective treatments for everyone, regardless of mutation."
Commenting on the long-term significance of the clinical trial results, Michael Boyle, M.D., senior vice president for therapeutics development, said: "Along with the excitement of potential immediate benefit for people with two F508del mutations, these results lay the groundwork for future potent combination therapies which include three different CFTR modulators. We believe these triple combinations will allow us to eventually increase the number benefitting from modulators to over 90 percent of people with CF."
Based on these results, Vertex announced plans to submit a New Drug Application to the FDA in the fall for review and potential approval of the combination treatment for people with two copies of the F508del mutation ages 12 and older as well as for individuals with one copy of the F508del mutation and a residual function mutation ages 12 and older.
For more information, see the Vertex press release.
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