Marta Figueiredo, PhD, science writer —

SPL84-23, an investigational antisense oligonucleotide (ASO), restored the levels and function of CFTR — the defective protein in cystic fibrosis (CF) — in lab-grown cells from patients carrying a common and disease-causing splicing mutation, a study reported. Based on these positive preclinical findings, SpliSense plans to launch a Phase 1/2…

Yap and Taz — two proteins involved in lung injury responses and regeneration — suppress the maturation of mucus-producing goblet cells and limit mucus levels in the lungs, according to a study of mice. These findings suggest that promoting Yap/Taz’s activity may be a potential therapeutic approach to limit the…

During adolescence and young adulthood, healthy siblings of cystic fibrosis (CF) patients have to find ways to achieve a balance between the illness of their brother or sister, family organization, and their own needs, a small study in Italy reported. Coping strategies included dampening emotional awareness, normalizing their life…

Cystic fibrosis (CF) patients with lower aerobic exercise capacity — assessed with a non-invasive cardiopulmonary exercise test — have a 4.5 times higher risk of future pulmonary exacerbations, according to a small 10-year study in Greece. Aerobic capacity, or VO2peak, is the maximal amount of oxygen a person’s…

Kalydeco (ivacaftor) safely and effectively improved lung function, growth, and nutrition in children and adults with cystic fibrosis (CF), while reducing rates of pulmonary flares and hospitalizations, according to a real-life study in France. These findings were consistent with those reported in previous clinical trials and real-world…

Cystic fibrosis (CF) carriers — and especially men with mutations in only one copy of CFTR, the gene associated with CF — are at a higher risk of developing a severe COVID-19 infection, one that carried a greater need for ventilation and could be quickly fatal, according to…

A mobile, dietary self-monitoring app used for six months to track food choices and manage pancreatic enzyme replacement therapy (PERT) helped children with cystic fibrosis (CF) to better meet disease-specific nutritional guidelines, a study shows. Still, the nutritional changes made were not sufficient to reach the standards of…

RCT223 and RTX0001, ReCode Therapeutics’ experimental RNA-based therapies for cystic fibrosis (CF), safely restored function to CFTR, the faulty protein in CF, in patient-derived lung cells. Delivered through the company’s non-viral platform — called the selective organ targeting (SORT) lipid nanoparticle (LNP) platform — the therapies were also…

Lower levels of certain volatile molecules in the breath of children and adults with cystic fibrosis (CF) can distinguish with good accuracy between those with and without Pseudomonas aeruginosa lung infection, a Dutch study shows. Notably, a reduction in the levels of a single molecule, called ethyl acetate, was…

The therapeutic benefits of the triple-combination medication Trikafta are mostly due to the effects of elexacaftor, a next-generation corrector of CFTR, the faulty protein in cystic fibrosis (CF), a study has found. These findings suggest that new combinations with elexacaftor may be even more effective than Trikafta. Trikafta,…