News

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for children with cystic fibrosis (CF) as young as one month old, who have at least one gating mutation in their CFTR gene that is responsive to the oral treatment. The agency’s approval was supported by data…

Children with cystic fibrosis (CF) who also have symptoms of attention-deficit/hyperactivity disorder (ADHD) may experience worse lung function, according to a new study from Israel and Spain. But the study also spotlighted a potentially far-reaching impact from ADHD on CF symptoms and care for children and adults…

The Phase 2 SPAN clinical trial of a new formulation of adrulipase in treating exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis (CF) has reached more than half of its target enrollment. Enrollment is ongoing at three U.S. sites, one each in Florida, Illinois, and Nevada. CF…

Vertex Pharmaceuticals has received a positive opinion from an arm of the European Medicines Agency on expanding the use of Orkambi (lumacaftor/ivacaftor) to children ages 1-2 who have two copies of the F508del mutation, the most common genetic defect in cystic fibrosis (CF). With the recommendation from…

From sharing personal stories to switching out Facebook cover photos, there are a variety of ways to participate in Cystic Fibrosis (CF) Awareness Month, set aside each May to acknowledge the CF community and call attention to its needs. CF is a genetic disease thought to affect some…

The U.S. Food and Drug Administration (FDA) has approved the expanded use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF) ages 2 to 5, who have at least one F508del mutation in the CFTR gene or a mutation that responds to Trikafta in laboratory studies.

A rare cell type that lines the airways, called pulmonary ionocytes, was successfully generated from stem cells derived from people with cystic fibrosis (CF), a study reports. Among all the cell types that line the airways, the recently discovered ionocytes, which make up about 1% of cells, have the…

Images taken via an ultrasound of the liver can be used to identify children with cystic fibrosis (CF) who may be at increased risk for advanced liver disease, a new study reports. “We have demonstrated that research-based [ultrasound] screening of children with CF can identify children with a high…

Variations in levels of certain regulatory RNA molecules in immune cells may help to explain why women and girls with cystic fibrosis (CF) tend to have a higher inflammatory response than do men and boys with the disease. “We and others have reported sex-differences in the course of CF,…

Cystic fibrosis (CF) patients who had fasting blood sugar (glucose) levels that rose more quickly throughout adolescence were more than three times as likely to have abnormal glucose tolerance (AGT) in early adulthood than their peers whose levels rose more slowly, a study found. AGT refers to elevated or…