Treatment with a triple combination — the new CFTR corrector VX-659, together with tezacaftor and Kalydeco (ivacaftor) — significantly improved lung function and was well-tolerated by cystic fibrosis (CF) patients, ages 12 and older, with at least one F508del mutation in two Phase 3 studies, according to Vertex Pharmaceuticals. This…
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Girls with cystic fibrosis (CF) reach puberty six to eight months later than healthy girls, about 16 percent of young females with CF encounter problems during sex, and 78 percent of these women would like to have children someday. These are among the key findings of an unprecedented survey conducted…
GEn1E Lifesciences has added to its development pipeline an investigational non-antibiotic, antibacterial agent for treatment of Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). The company established an exclusive option agreement with the University of Maryland, Baltimore (UMB) to license patent rights over the MUC1-ecto-domain (MUC1-ED) compound, currently…
A protein toxin called Tse7 is used by Pseudomonas aeruginosa to kill neighboring bacteria, and may help explain why lung infections in cystic fibrosis patients are often dominated by a single P. aeruginosa strain, a new study reports. The research, “The Pseudomonas aeruginosa T6SS-VgrG1b spike is topped by…
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…
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