News

The CFTR modulator therapy Kalydeco (ivacaftor) can safely be given to babies with cystic fibrosis (CF) as young as 4 weeks old, according to data from a Phase 3 clinical trial. The findings were the basis of the U.S. Food and Drug Administration (FDA)’s decision to expand…

Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) led to gains in body mass among cystic fibrosis (CF) patients that were largely driven by increases in various types of fat and low-density muscles, a study found. High-density muscle, which is stronger than its lower-density counterparts, didn’t change with the treatment, sold as…

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…

The Cystic Fibrosis Foundation (CFF) on April 30-May 1 will present ResearchCon, a free virtual event for patients, caregivers, scientists, and clinicians that will explore research advancements and the future of cystic fibrosis (CF). The event, created with the CF community, will include keynote addresses, presentations, roundtable…

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…

In lab studies, researchers have identified rare mutations in the CFTR gene — which are the cause of cystic fibrosis (CF) — that are not currently eligible for CFTR modulator therapies but might be treatment responsive. The researchers hope the findings will provide guidance to developer Vertex Pharmaceuticals…

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…