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Note: This story was updated Nov. 9 to note that experiments done with Sionna’s CFTR modulators used a different investigational NBD1 stabilizer than SION-638. SION-638, a first-in-class small molecule that is being developed by Sionna Therapeutics, improved the stability and functionality of the CFTR protein  in the most common…

Exposure to tobacco smoke may reduce the effectiveness of Kalydeco (ivacaftor), a CFTR modulator that is widely approved to treat cystic fibrosis (CF) caused by specific mutations. That’s according to data shared by Gabriela Oates, PhD, at the 2022 North American Cystic Fibrosis Conference, taking place Nov. 3–5…

A novel scoring system may be used to classify the severity of lung disease among children with cystic fibrosis (CF) in their first years of life. Leslie Huang, a medical student at the University of Wisconsin, discussed the system at the 2022 North American Cystic Fibrosis Conference in Philadelphia,…

Treatment with Trikafta continues to be safe and well-tolerated, benefiting people with cystic fibrosis (CF) ages 12 and up who have been on treatment for almost three years. But benefits may also extend to children as young as 2. That’s according to data that will be shared at…

First Wave BioPharma is teaming up with Rho for a Phase 2 clinical trial to test their new adrulipase formulation, designed as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). “We are eager to…

Twice-weekly sessions of airway clearance physiotherapy over eight weeks led to clinically meaningful reductions in respiratory symptoms in people with cystic fibrosis (CF) in a small clinical trial. These preliminary findings, based on patient-reported measures, support the need for further studies to determine the most effective airway clearance technique,…

Researchers in Oregon have developed lipid nanoparticles that can be inhaled and effectively deliver messenger ribonucleic acid (mRNA) into the lungs of mice, triggering the cells to produce the defective protein in cystic fibrosis (CF). “Inhaled [lipid nanoparticles] resulted in localized protein production in the mouse lung without toxicity,…

For a small group of people with cystic fibrosis (CF), one year of treatment with Orkambi (ivacaftor/lumacaftor) improved bone health and stabilized pancreatic function, nutritional status, reproductive hormone levels, and lung function. These findings from a recent study in Israel demonstrate the use of Orkambi, which targets the…

Treatment with Orkambi (lumacaftor/ivacaftor) for six months was found to improve exercise endurance in three adults with cystic fibrosis (CF), a new study showed. Moreover, by the end of the study, all three men experienced less leg discomfort and less dyspnea, or shortness of breath. For two patients,…

The incidence of colorectal cancer (CRC) in people with cystic fibrosis (CF) was five times higher than in those without CF, after adjusting for age, in a large-scale English population study. CFTR gene mutations, the underlying cause of CF, occurred more frequently than expected, suggesting an association between…