Margarida Maia, PhD,  science writer—

Note: This story was updated Nov. 18, 2021, to correct information about Pulmozyme’s administration method. Bronchitol (mannitol) plus Pulmozyme (dornase alfa) is better than Pulmozyme alone to help loosen mucus in the lungs in children with cystic fibrosis (CF), and this seems to translate into better lung function. The…

The Cystic Fibrosis Foundation (CFF) is partnering with Pioneering Medicines, an initiative of Flagship Pioneering, to support the development of various types of gene therapy for cystic fibrosis (CF). The foundation is ready to invest up to $110 million to move therapy candidates into clinical testing, starting with an…

MRI scans may help to reveal subtle changes in certain areas of the brain among people with cystic fibrosis (CF), a study found. The affected areas are involved in mood and cognition, which were found to be altered relative to people without the genetic disease. “CF patients exhibited significant…

A Phase 1/2 clinical trial of an aerosol gene therapy candidate, 4D-710, in people with cystic fibrosis (CF) has been cleared by U.S. Food and Drug Administration (FDA) and is expected to start this year, its developer, 4D Molecular Therapeutics, announced. The trial is designed as an open-label, dose-escalation…

Pharmacy benefits and services company Elixir has chosen Maxor Specialty Pharmacy to be its network’s exclusive provider of medications for cystic fibrosis (CF). Maxor Specialty specializes in rare diseases like CF and is one of four pharmacies in the U.S. to have access to all medications approved for cystic fibrosis,…

Poor bone density is increasingly evident in older cystic fibrosis (CF) patients, and may be linked to an inflammation biomarker known as interleukin 8 (IL-8), a small study in Canada suggests. Bone disorders can affect 24% of adults with CF, the study noted, adding that the Cystic Fibrosis Foundation…

Cystic fibrosis transmembrane conductance regulator (CFTR), the protein that is mutated in cystic fibrosis (CF), is not as rigid a structure as previously thought, but rather one that may transition between shapes, a molecular study has found. Under certain conditions — such as the most common CF-causing mutation, F508del…

Kaftrio, marketed as Trikafta in the U.S., may be effective in people with cystic fibrosis (CF) who have one F508del mutation plus one other unknown mutation in the disease-causing gene, a study in cells taken from three such patients suggests. The results indicate that the combination therapy, which…

Symdeko (tezacaftor/ivacaftor) favors weight gain, shortens hospital stays, and reduces the number of days on antibiotics in patients with cystic fibrosis (CF), a real-world study of its use at a Scottish clinic reported. However, no significant benefits in patients’ lung function were seen over about a year of…

Sildenafil improves how muscles work when patients with cystic fibrosis (CF) exercise and this translates into better exercise capacity, a new study suggests. The study, “Exercise intolerance in cystic fibrosis: importance of skeletal muscle,” was published in the journal Medicine & Science in Sports &…