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The Cystic Fibrosis Foundation is aggressively pursuing potential treatments for people with CF who have nonsense and rare mutations who will not benefit from drugs known as modulators, which correct the malfunctioning cystic fibrosis transmembrane conductance regulator (
Since 2011, the Foundation has awarded $2.5 million across 35 different projects to improve cystic fibrosis newborn screening across the country.
Newborn screening (NBS) is a program run by each state to identify babies born with certain health conditions, including cystic fibrosis. Although a sweat test should ultimately be done to rule out or confirm a CF diagnosis, NBS can help you and your health care providers take immediate steps to keep your child as healthy as possible.
Dr. Dorothy Andersen first wrote about cystic fibrosis in 1938, and our understanding of the disease has significantly evolved since. There are still many misconceptions about what CF looks like today, from the person who lives with the disease to how it manifests.
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
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You may be waiting for a transplant for a long time. While you're waiting, there are some things you will have to do in addition to your normal routine, to ensure you remain healthy and eligible for transplant.