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 Cystic Fibrosis Foundation today applauds the Food and Drug Administration's approval of Kalydeco™ (ivacaftor; previously known as VX-770), a major advance in the search for a cure for cystic fibrosis.
Published on January 31, 2012
The drug was developed by Vertex Pharmaceuticals Inc., with scientific, clinical and significant funding support from the Cystic Fibrosis Foundation.
The FDA approved Kalydeco (kuh-LYE-deh-koh) for a segment of the CF population, those ages 6 and older with the G551D mutation of cystic fibrosis. The drug is taken in pill form.
“Today marks an important milestone in our journey to find a cure for cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “Kalydeco addresses the underlying cause of CF, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people living with CF.”
The G551D mutation is present in roughly 4 percent of the CF patient population in the United States. In people with this mutation, a defective protein caused by CF moves to its proper place at the surface of the cell but does not function correctly. Instead, the defective protein acts like a locked gate, preventing the proper flow of salt and fluid in and out of the cell.
Kalydeco helps unlock that gate and restore the function of the defective protein. The drug dramatically improves lung function, lowers sweat chloride levels and helps patients gain weight -- all key clinical indicators of CF.
The CF Foundation played a significant role in the research and development of this groundbreaking new drug. “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development,” said FDA Commissioner Margaret A. Hamburg, M.D., in a news release issued by the FDA.
While Kalydeco represents a breakthrough for patients with the G551D mutation, it also represents hope for all people with CF, according to Preston W. Campbell, III, M.D., the CF Foundation's executive vice president for medical affairs. “Our organization's mission is to find a cure for cystic fibrosis, and we won't stop our work until we reach our goal for all people living with the disease.”
The vast majority of individuals with CF in the United States -- nearly 90 percent -- have a different mutation, called Delta F508.
Developing therapies for patients with the Delta F508 mutation is among the current research priorities of the CF Foundation. Progress toward this goal includes an ongoing Phase 2 clinical trial of Kalydeco in combination with another potential therapy, VX-809, in people with the Delta F508 mutation. Results from the first part of this Phase 2 trial were positive. The second part of the study is now under way.
In addition, the CF Foundation has significantly expanded its research investments with other leading pharmaceutical companies, including Genzyme and Pfizer, to accelerate the discovery and development of new drugs that will help more CF patients.
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