Among young children with cystic fibrosis (CF), starting the approved oral therapy Orkambi (ivacaftor/lumacaftor) led to favorable changes in nutrition and growth status during the first six months of treatment, according to a small real-world study. Orkambi, first approved in the U.S. in 2015 and now indicated for…
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Danish children with cystic fibrosis (CF) born between 2000 and 2022 showed noticeable gains in growth during their first five years of life, with those born after 2016 seeing the most progress in reaching normal growth standards, a study shows. This is likely due to newborn screening programs (NBS)…
Vertex Pharmaceuticals and England’s National Health Service (NHS) have reached an agreement that allows all current and future eligible cystic fibrosis (CF) patients to access three of the company’s approved CFTR modulators at low or no out-of-pocket cost. Kaftrio (elexacaftor/tezacaftor/ivacaftor, sold as Trikafta in the U.S.), Symkevi (tezacaftor/ivacaftor,…
Children with cystic fibrosis (CF) who are being treated with Symdeko (ivacaftor/tezacaftor) or Orkambi (ivacaftor/lumacaftor) experience few to no symptoms of chronic rhinosinusitis and maintain a normal sense of smell, a study in Denmark found. Although small, the study suggests that not only Trikafta, a triple…
Despite some similarities, the lung composition of microbes and their antimicrobial resistance profiles differed between people with bronchiectasis and cystic fibrosis (CF) and those with non-CF bronchiectasis, a study found. The findings suggest “the need for customized management strategies for each disease,” the researchers wrote in the study, “…
CFTR modulators may help control glucose (sugar) levels in the blood of people with cystic fibrosis (CF), according to researchers in Israel, who called for regular oral glucose tolerance testing to adjust insulin dosing in diabetes and keep glucose from dropping too low. The study, “Long-Term therapy with…
Additional mutations in the SLC26A9 gene among cystic fibrosis (CF) patients with two copies of F508del — the most common CF-causing mutation in the CFTR gene — were associated with more rapidly declining lung function. These findings from a study further buttress the idea that variation in genes other…
A lung-targeted gene-editing therapy designed using technology from ReCode Therapeutics was able to successfully correct a disease-causing CFTR gene mutation in lung cells derived from cystic fibrosis (CF) patients, according to a recent proof-of-concept study. The therapy, developed using ReCode’s Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), was…
Low doses of ETD001, an inhaled medication being develop by Enterprise Therapeutics to treat cystic fibrosis (CF), acted for longer than expected and helped to clear mucus from the airways of sheep in a study. Equivalent doses also were well tolerated in an earlier Phase…
During pulmonary exacerbation in cystic fibrosis (CF), bacterial communities in the lungs tend to change in one of two general patterns, and better understanding these patterns may help predict how patients will respond to antibiotic treatments. That’s according to the study, “Microbial community organization designates distinct…
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