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There is wide clinical variety in the presentation of individuals who carry one F508del mutation and one 5T;TG12 mutation in the CFTR gene, an Italian study highlights. Its researchers stressed the need to monitor individuals with this genotype, since there are not currently accurate ways to predict who is at highest risk…

ReCode Therapeutics’ investigational aerosolized RNA-based therapies for cystic fibrosis (CF) restored CFTR function, the defective protein in CF, in patient-derived lung cells. Delivered by the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform, the therapy showed no signs of toxicity in epithelial cells that line the airways.

LungFit GO, inhaled nitric oxide (NO) that can be self-administered at home, was well tolerated and improved various quality of life measures, according to recent data from a pilot study that evaluated the experimental treatment in adults with or without cystic fibrosis (CF). The ongoing study involves people…

For the first time, bacteriophages — viruses that kill bacteria — eliminated an antibiotic-resistant mycobacterial lung infection in a man with advanced cystic fibrosis (CF) lung disease, a study reported. As a result, the patient was able to undergo a successful lung transplant after one year of bacteriophage treatment.

Lab-edited transfer RNA (tRNA) molecules revived the production of a protein that’s lost or faulty in cystic fibrosis patients’ cells that carry mutations in the protein’s coding gene, a study found. They did this by helping the cell machinery read through the mutations that would cause protein production to…

A small compound called c407 restored the function of CFTR — the defective protein in cystic fibrosis (CF) — and its associated chloride flow in mice carrying F508del, the most common CF-causing mutation, a study shows. These benefits were associated with a stabilization of CFTR’s folding and its increased transport to the…

The presence of lung disease was low among infants with cystic fibrosis (CF) and was not associated with respiratory infections, yet respiratory symptoms, like cough or wheezing, may be a sign that lung disease is progressing, according to a recent study. “Respiratory symptoms in infants with CF deserve attention…

The frequency of cystic fibrosis-associated liver disease (CFLD) has increased in the past two decades and is linked to several simultaneous health complications, a U.S. study shows. CFLD was also found to be independently associated with a higher risk of mortality and increased healthcare resource use in children hospitalized due…

Cystic fibrosis (CF) patients with non-tuberculous mycobacteria (NTM) lung infection show significant changes in airway metabolites relative to those without such infections, a study shows. Metabolites are intermediate or end products of cellular metabolism, and some of these altered metabolites play roles in immune responses and bacterial growth. Combining…

Infants with cystic fibrosis (CF) who are infected with Pseudomonas aeruginosa in their first six months are at heightened risk of having impaired lung function by the time they are preschool age, a new study reports. The study also showed that lung function measurements and CT scan imaging in…