Marisa Wexler, MS,  senior science writer—

While Kaftrio — sold as Trikafta in North America — can improve lung function in children with cystic fibrosis (CF), treatment effects are unlikely to be as dramatic in real-world practice as those reported in clinical trials. Rather, for eligible children without substantial lung problems, Kaftrio’s use can…

Mutations that cause cystic fibrosis (CF) lead to biochemical changes in lung cells that make it easier for infectious bacteria to stick to the cells, a new study reports. Findings notably suggest that treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) did not reverse these changes, which may help to explain why…

The U.S. Food and Drug Administration (FDA) has given the go-ahead for a Phase 2 trial testing the inhalation therapy SPL84 in people with cystic fibrosis (CF) caused by a specific mutation called 3849+10 kb C-to-T. Therapy developer Splisense welcomed the FDA’s clearance of the clinical trial, and…

For adults with cystic fibrosis (CF), the symptoms with the biggest impact on quality of life include pain, fatigue, shortness of breath, and mental health challenges, leading researchers to recommend routine screening to assist CF patients who could benefit from support. “Screening for prevalent symptoms that affect quality of life…

Bacteria in the airways of people with cystic fibrosis (CF) can interfere with the growth of Pseudomonas aeruginosa by secreting acetic acid, a main ingredient in vinegar. That’s according to “Airway commensal bacteria in cystic fibrosis inhibit the growth of P. aeruginosa via a released metabolite,”…

4D Molecular Therapeutics (4DMT) is planning a Phase 3 clinical trial that, if results are positive, will be the basis of applications seeking approval of the company’s inhaled gene therapy 4D-710 as a treatment for people with cystic fibrosis (CF) who can’t take CFTR modulators. 4DMT…

Sionna Therapeutics has raised $182 million in financing to advance the development of small molecules designed to restore CFTR protein function by binding to a specific part of the protein called the first nucleotide-binding domain, or NBD1. Cystic fibrosis (CF) is caused by mutations in the gene…

A lipid nanoparticle designed to deliver genetic therapy to lung cells could be used as a platform to treat lung-related diseases like cystic fibrosis (CF), a mouse study found. Researchers developed a way to identify lipid nanoparticles — fatty molecules that help deliver genetic material to cells — that…

A persistently stuffy nose is a big concern for people with cystic fibrosis (CF) who have chronic nose inflammation, but loss of smell isn’t as significant a worry, according to a new study that used the SinoNasal Outcome Test (SNOT-22). “Nasal congestion and post-nasal discharge were top priorities reported…

In the years after Kalydeco (ivakaftor) became available, the use of pancreatic enzyme replacement therapy (PERT) fell among people with cystic fibrosis (CF) in the U.S. who were eligible for the therapy. That’s according to “Pancreatic enzyme prescription following ivacaftor licensing: A retrospective analysis…