News

The European Commission has approved Kalydeco (ivacaftor), by Vertex Pharmaceuticals, to treat infants as young as four months old with cystic fibrosis (CF) caused by certain mutations. The decision to extend Kalydeco’s label to younger babies, weighing at least 5 kg (about 11 lbs) and with relevant mutations, follows…

The Cystic Fibrosis Foundation (CFF) has awarded funding to enGene to support its work on a non-viral and inhalable gene therapy that could deliver a working copy of the CFTR gene to the lungs of people with cystic fibrosis (CF). “Today’s announcement reflects yet another milestone on our path…

The U.S. Food and Drug Administration (FDA) has approved the dry inhalation powder Bronchitol (mannitol) as a maintenance treatment to be used along with other therapies for improving lung function in adults with cystic fibrosis (CF). Bronchitol is currently approved and marketed in Switzerland, Australia, Italy,…

Pseudomonas aeruginosa mutate into a mucoid form in the lungs of people with cystic fibrosis (CF), creating biofilms that render these bacteria more resistant and more damaging, a study reported. Its researchers suggest a way of studying P. aeruginosa cells to identify treatments…

Envara Health has launched Encala (formerly Lym-X-Sorb), a nutritional supplement designed to improve fat and nutrient absorption in people with cystic fibrosis (CF) and pancreatic insufficiency. Encala is indicated for adults and children older than 1 who have started eating toddler or family table food. According…

The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause  cystic fibrosis (CF), spinal muscular atrophy (SMA), and fragile X syndrome be open to reimbursement for couples planning or in the early stages of pregnancy. Its favorable opinion…

Machine learning, a method of data analysis, could improve disease prediction and bring precision medicine for cystic fibrosis (CF) one step closer to reality, according to researchers at the University of Cambridge, in the U.K., who have developed novel artificial intelligence (AI) technologies to allow for clinical…

Oral lenabasum failed to significantly reduce the frequency of pulmonary exacerbations in people with cystic fibrosis (CF) treated in a Phase 2 trial, but new analyses suggest this failure could be attributed, at least in part, to differences among the patients that confounded results. The investigational therapy showed some…

Eloxx Pharmaceuticals presented preclinical safety and efficacy data supporting ELX-02, its investigational treatment for cystic fibrosis (CF) caused by nonsense mutations that is also being evaluated in two enrolling, Phase 2 trials. The data were presented as two posters at this year’s virtual North American Cystic Fibrosis…

The National Board for Respiratory Care (NBRC) has launched “CHOOSE ONE: Every Breath Counts Campaign,” a fundraising initiative intended to protect and improve the lives of people who need respiratory care, such as those with lung diseases like cystic fibrosis (CF). This year, more than $200,000 will…