News

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…

30 Technology has sold the rights to its antimicrobial treatment platform for certain uses, such as for burns and acute wounds, to Convatec. 30 Technology will retain the rights to its wound care platform in other indications, including respiratory diseases such as cystic fibrosis (CF), and will be…

SPRIM Global Investments (SGI) has committed $4.5 million to initiate Spexis’ Phase 3 COPILOT clinical trial to test the antibiotic ColiFin in people with cystic fibrosis (CF) who have chronic Pseudomonas aeruginosa lung infections. The trial will test once or twice daily dosing of ColiFin (inhaled colistimethate sodium),…

Monitoring the concentration of antibiotics in the blood — a clinical practice called therapeutic drug monitoring — was linked to fewer pulmonary exacerbations and less lung function decline in people with cystic fibrosis (CF), a small study reports. The study, “Therapeutic drug monitoring in cystic fibrosis…