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.
PTC Therapeutics has discontinued development of ataluren as a potential treatment for people with cystic fibrosis caused by a nonsense mutation. The Cystic Fibrosis Foundation is supporting efforts to pursue treatments for people with CF who have a nonsense, splicing or other rare mutations.
Published on March 3, 2017
The drug company PTC Therapeutics announced on March 2 that it has discontinued the development of ataluren as a potential treatment for people with cystic fibrosis. Ataluren was an investigational therapy for CF caused by nonsense mutations, which prevent the production of a normal cystic fibrosis transmembrane conductance regulator (CFTR) protein. A dysfunctional or nonexistent CFTR protein disrupts the salt and water balance in the lungs and other tissues and leads to the thick, sticky mucus associated with CF.
The company reported that ataluren did not show significant improvement in lung function or enough of a decrease in exacerbations in its most recent Phase 3 clinical trial. The trial, which began in 2015, was for people with at least one copy of a nonsense mutation who were not taking inhaled aminoglycosides, such as tobramycin.
Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Foundation, had provided support for the development of ataluren.
"This result is disappointing for the entire CF community. We stand together until all people with CF have the transformative therapies they need,” said Preston W. Campbell, III, M.D., president and chief executive officer of the Cystic Fibrosis Foundation. “We are aggressively pursuing research into a variety of approaches that may benefit people with nonsense mutations.”
The Foundation is supporting multiple efforts to pursue treatments for the 10 percent of people with CF who have a nonsense, splicing or other rare mutation for which there is no current therapy that targets the root cause of their disease.
CFFT is spearheading a Nonsense and Rare Mutations Research and Therapeutics Initiative, which is expected to grow significantly. The initiative consists of more than 60 collaborative projects with an array of academic programs and pharmaceutical companies around the world.
The initiative includes:
Research into nonsense and rare mutations is also ongoing at the recently expanded CFFT research lab, which is focused on identifying candidate therapies for nonsense mutations. Efforts focused on nonsense mutations are also underway through a program led by the nonprofit research organization Southern Research, in collaboration with the University of Alabama (UAB) and CFFT.
CFFT also will hold an international workshop to promote the further development of laboratory cell models to better predict the response of new therapies in people with CF.
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