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 celebrates the FDA's approval today of the lumacaftor/ivacaftor combination drug (Orkambi™) as an important advance for the cystic fibrosis community. Orkambi targets the underlying cause of the disease in people with two copies of the most common CF gene mutation.
Published on July 2, 2015
The FDA approved the drug for people with CF ages 12 and older who have two copies of the F508del mutation, representing 8,500 people or nearly one-third of the CF population in the United States. Nearly half of individuals with CF have two copies of this mutation and could eventually benefit from the therapy.
Orkambi was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific and funding support from the CF Foundation.
“We applaud the FDA for its swift approval of Orkambi,” said Robert J. Beall, president and CEO of the CF Foundation. “It is our hope that everyone who is prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives.”
Beall continued: “We are proud of the important role that the Foundation played in the research and development of both ivacaftor and lumacaftor, including initiating the first efforts that led to the discovery and development of these small molecules.”
Orkambi improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. It is the second drug approved by the FDA that targets the underlying cause of CF. Ivacaftor (as a single therapy) was the first.
Cystic fibrosis is a life-threatening genetic disease that causes serious lung infections and premature death. It affects 30,000 children and adults in the United States and 70,000 people worldwide. People with the disease inherit a defective gene that causes a buildup of thick mucus in the lungs, pancreas and other organs.
In people with two copies of the F508del mutation, a series of problems prevents a protein, called CFTR, from achieving the correct shape and reaching the cell surface, where it is needed to help regulate the flow of salt and fluids in and out of the cells of the lungs and other organs.
Orkambi combines lumacaftor and ivacaftor to treat these problems with a two-step approach. Lumacaftor helps move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs of people with CF.
“While the approval of Orkambi is a meaningful step forward, we won't lose sight of the promises we've made to all people with CF and their families,” said Preston W. Campbell, III, M.D., executive vice president for medical affairs of the CF Foundation. “We will continue to fight the disease from every angle, exploring all promising avenues of research, including those that are pioneering new technologies to repair the defective CF gene, so that one day all people with CF will no longer have the disease.”
Orkambi is the latest example of the CF Foundation's venture philanthropy model, through which it has invested hundreds of millions of dollars to help develop CF drugs and therapies. This approach has led to tremendous advances in life expectancy for people with CF, which has doubled in the past 30 years. Nearly every CF drug available today was made possible because of Foundation support.
In addition to supporting research programs with Vertex, the CF Foundation invests in the CF research programs of many leading pharmaceutical companies, including Pfizer, Genzyme and Shire, to accelerate the discovery and development of new drugs that will help more people with CF.
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