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.
Vertex Pharmaceuticals Inc. announced today that it has submitted a new drug application to the U.S. Food and Drug Administration (FDA) for approval of the combination of ivacaftor (Kalydeco™) and lumacaftor. The potential drug is for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation, the most common CF mutation.
Published on November 5, 2014
The company has asked the FDA for priority review of the combination therapy, which, if granted, could shorten the review timeframe from approximately 12 months to 8 months. The FDA grants priority review for several reasons, including when a potential drug is considered a major treatment advance.
The treatment is the first to combine two drugs into a single pill to address the underlying genetic cause of the disease in those with two copies of the F508del mutation. About 50 percent of people with CF in the United States have two copies of the F508del mutation and 40 percent have one copy.
“The CF Foundation is very pleased to see that this combination treatment is moving forward with possible approval in 2015,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This is an exciting step forward in our efforts to speed development of new and effective therapies for all people with CF.”
Vertex has also submitted an application to the European Medicines Agency for approval of the drug for people with two copies of the F508del mutation in the European Union. The combination treatment has already been granted accelerated assessment in the European Union.
Approximately 8,500 people with CF ages 12 and older in the United States and 12,000 in Europe have two copies of the F508del mutation.
Results released earlier this year from late-stage clinical trials of the combination treatment in those with two copies of F508del showed that those who received the drug had significant improvement in lung function and other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations. The combination treatment was generally well tolerated by participants who took the drug, with few adverse events reported.
Vertex plans to begin a Phase 3b clinical trial of the potential combination drug in people ages 12 and older with two copies of the F508del mutation who have severe lung disease and may benefit from the treatment prior to its potential approval. The trial will enroll a limited number of people in the first quarter of 2015.
People with CF and their families who have questions about the Phase 3b clinical trial may contact Vertex Medical Information (in the United States) at 1-877-634-8789 or firstname.lastname@example.org, or (outside the United States) email@example.com.
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