News

Rice University researchers are using a virus that infects moths and magnetic nanoparticles to develop a new vehicle for the delivery of CRISPR/Cas9, a genome-editing tool, with high efficiency and tissue specificity in vivo. CRISPR/Cas9 has gained attention as a potential tool to correct genetic defects linked to diseases…

The European Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Orkambi (lumacaftor/ivacaftor) as a treatment for children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals announced. CHMP’s positive opinion will be taken into consideration by the European Medicines Agency, which will…

A new test was able to assess various properties of the mucus that accumulates in the lungs of people with cystic fibrosis and other pulmonary diseases, and may help to find treatments — or a combination of treatments — for CF patients that can make coughing a more viable way…

Ultrasound may predict liver disease in young cystic fibrosis (CF) patients, according to the results of a clinical trial. Researchers compared liver patterns from young CF patients, and found that those with non-uniform patterns on ultrasound are more likely to develop liver scar tissue. Results from the the Cystic Fibrosis…

Antibacterial Avycaz (ceftazidime-avibactam) showed significant in vitro efficacy against multidrug-resistant Pseudomonas aeruginosa collected from the sputum of cystic fibrosis (CF) patients, according to a recent study. The study, “Multidrug-resistant Pseudomonas aeruginosa from sputum of patients with cystic fibrosis demonstrates a high rate of susceptibility to ceftazidime–avibactam,” was…

Cystic fibrosis (CF) patients with liver disease do not experience greater lung function decline compared with CF patients without liver presentation, a study reports. The study, “Analysis of a large cohort of cystic fibrosis patients with severe liver disease indicates lung function…

Ten of 19 cystic fibrosis (CF) patients given antibiotics to manage pulmonary exacerbations did not achieve high enough blood concentrations of the antibiotic to sustain a therapeutic effect, results of single-site study show. This failure may be contributing to the development of antibiotic-resistant pulmonary infections, and to a worsening of…

Patients with cystic fibrosis (CF) who carry certain mutations in the HFE gene, which are also behind an iron overload disorder, have poorer lung function that declines faster and a higher risk of CF-related diabetes (CFRD) and intestinal blockage,…

The Anton Yelchin Foundation has donated $1 million to the University of Southern California (USC) Center for Cystic Fibrosis – Adult Care at Keck Hospital to thank the center for its efforts in managing the healthcare of the late American actor. Yelchin, who had cystic fibrosis (CF), was cared…

Positive data from preclinical and Phase 2a studies evaluating the investigational cystic fibrosis (CF) treatment RPL554 were presented at the 2018 North American Cystic Fibrosis Conference (NACFC) in Denver, Colorado (Oct. 18-20). RPL554, developed by Verona Pharma, is an inhaled potential therapy for CF and chronic obstructive pulmonary disease (COPD).