Margarida Maia, PhD,  science writer—

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 &…

Vitamin D intoxication may occur in people with cystic fibrosis (CF) due to accidental errors made by pharmacists in preparing prescriptions, a study has found. Indeed, some 13 patients in the study were taking a dose of vitamin D that was nearly 70 times higher than the prescribed one,…

People with cystic fibrosis (CF) generally are living longer and healthier lives than in the recent past. Now, researchers will conduct a five-year study to look into ways to further increase and improve the lifespan of people with the disease. The idea behind the study comes from a collaborative…

To mark its 10th anniversary, Claire’s Place Foundation has launched a website that seeks to connect the community of people affected by cystic fibrosis (CF). “Our new website is a supportive forum for inspiration as well as a resource to learn about developments in CF, our programs and…