Patricia Inácio, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has granted priority review to Vertex’s new drug application for its triple combination — elexacaftor (VX-445) plus tezacaftor and Kalydeco (ivacaftor) — for cystic fibrosis (CF) patients who cannot use the company’s other disease-modifying treatments or don’t benefit as intended.

Trained sniffer dogs are able to detect Pseudomonas aeruginosa, the most common bacteria found in people with cystic fibrosis (CF), in liquid cultures even at very low concentrations, a study shows. This potentially could be useful for detecting infections earlier in CF patients — and without the need…

Lung transplants are the ultimate therapeutic strategy for many people with cystic fibrosis (CF) and other chronic lung diseases, but they are scarce due to the severe shortage of donor organs, which currently is the only way of obtaining a lung. Research in the lab of Laura Niklason, MD,…

A minimally invasive imaging device that generates high-resolution images of nasal airways, including the cilia and mucus, allows researchers to visualize changes at the cellular level that underlie cystic fibrosis (CF) in patients. The data confirms that mucus dehydration and abnormalities of the airway’s epithelia, as well as…

Using the CRISPR gene editing tool, a team of researchers was able to permanently correct two mutations in the CFTR gene — mutations that cause cystic fibrosis (CF) — in CF patient-derived cells. The researchers showed that the corrected CFTR gene was functional and working normally in mini-intestines called…

Treatment for three years with Kalydeco (ivacaftor) reduces the risk of lung infection by serious pathogens — including Pseudomonas aeruginosa and Staphylococcus aureus — in people with cystic fibrosis (CF), a U.K. study reports. The study, “Ivacaftor Is Associated with Reduced Lung Infection by Key Cystic Fibrosis…

Treatment with Kalydeco (ivacaftor) improved lung function, exercise capacity, and sweat chloride concentration in cystic fibrosis (CF) patients with severe lung disease, a small retrospective analysis shows. The study, “Effectiveness of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations,” was published in the journal Pediatric Pulmonology. Mutations…

Long-term use of inhaled antibiotics, given to treat lung infections caused by the bacteria Pseudomonas aeruginosa  in people with cystic fibrosis (CF), can for young children raise the risk of a new infection by Aspergillus fungi, a study suggests. The study, “Pseudomonas…

Treatment with Orkambi significantly improves exercise tolerance in adult cystic fibrosis patients with severe disease as early as 4 weeks after treatment — as shown by results of the six-minute walk test (6MWT), a study found. The findings support the use of exercise tolerance as an important…

Data from Phase 1 studies in healthy people and preclinical work indicate that ELX-02 may help to treat cystic fibrosis (CF), Eloxx Pharmaceuticals, its developer, reported. The therapy was well-tolerated in single- and multiple-ascending dose studies in those clinical trials, and raised levels of the crucial CFTR protein in…