cystic fibrosis

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Treatment with Kalydeco (ivacaftor) alone improved lung function and quality of life in a cystic fibrosis patient carrying both a therapy-responsive and a non-responsive mutation to treatment, a case study reports. The case study, titled “Positive clinical outcomes following ivacaftor treatment in a cystic fibrosis patient with the…

High levels of the peptide copeptin are associated with increased severity of cystic fibrosis (CF) in children, a study shows. These results suggest that copeptin can be used as a biomarker to identify patients who could benefit from more aggressive treatment. The study, titled “Evaluation of…

The Government of Quebec, in Canada, has approved a loan of $2.7 million Canadian (about $2.05 million U.S.) to Laurent Pharmaceuticals to help the company conduct a Phase 2 clinical trial to evaluate its investigational oral candidate LAU-7b for…

Researchers in Israel and the U.S. have developed a new technology that can be used to unclog and eliminate mucus secretions from the airways of patients with various respiratory diseases, including cystic fibrosis (CF). The announcement was made by BGN Technologies, the technology company of Ben-Gurion University…

To heighten cystic fibrosis (CF) awareness and raise funds for research, care and advocacy, Cystic Fibrosis Canada (CFC) has launched its fourth annual year-end campaign. This year’s effort, which began Nov. 28 and runs through Dec. 31, is called “Wish.” The nonprofit will also match all pledges up to…

The therapeutic triple combo elexacaftor (VX-445), tezacaftor, and ivacaftor leads to marked improvements in lung function and quality of life in people with cystic fibrosis (CF) carrying the F508del mutation, data from a Phase 3 trial show. The findings were reported in a study, “Efficacy and…

When it comes to lung transplants, the Cystic Fibrosis Foundation (CFF) admits that for a long time the issue was shrouded in confusion, frustration, and lack of information. But that’s changed substantially in recent years, says Albert Faro, MD, head of the nonprofit’s Advanced Lung Disease Initiative. “In…

Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days of treatment with intravenous antibiotics could help predict treatment response in cystic fibrosis patients experiencing pulmonary exacerbations, a study has found. The study, “Identification of novel blood biomarkers of treatment response…

Researchers have discovered a stress response mechanism that allows Pseudomonas aeruginosa, a bacteria highly resistant to antibiotics that often causes lung infections in patients with cystic fibrosis (CF), to evade the action of antibiotics and to survive. The findings were reported in a study, “PQS Produced…