News

Vertex Pharmaceuticals announced a multiyear partnership with Affinia Therapeutics to develop gene therapies for cystic fibrosis (CF) and other genetic disorders. Gene therapy seeks to replace a malfunctioning gene with a working version. In people with CF, in particular, a gene therapy would deliver a healthy copy of the…

Supplemental estrogen, most commonly taken for contraception, is associated with poorer bone mineral density in people with cystic fibrosis (CF), a new study suggests. The study, “Oral ethinyl estradiol treatment in women with cystic fibrosis is associated with lower bone mineral density,” was published in the …

FFF Enterprises and Bionews Services, publisher of this website, announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone.

A measure called the lung clearance index (LCI) can predict low blood oxygen levels during sleep in people with cystic fibrosis (CF) better than conventional assessments of lung function, a study suggests. The study, “Lung clearance index evaluation in detecting nocturnal hypoxemia in cystic fibrosis patients:…

A panel of experts has released a series of recommendations for carers of children with chronic conditions, such as cystic fibrosis (CF), asthma, congenital heart disease, and diabetes, about how to manage these conditions from home. The new guidelines come at a much-needed time, as they may…

Vertex Pharmaceuticals announced that a reimbursement program covering Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor combo) will be opening for eligible cystic fibrosis patients (CF) living in Switzerland. The program is part of a recent agreement reached between the company and the Swiss Federal Office of Public Health (FOPH) and…

Different types of Staphylococcus aureus can be found persistently in the airways of cystic fibrosis (CF) patients, but this bacteria population is influenced by age and prior antibiotic treatment, a new study says. …

Problems with a protein called TAS2R38 are linked to a greater risk of severe sinonasal disease and Pseudomonas aeruginosa lung infections early in life in people with cystic fibrosis (CF), a study reported. These findings may help to identify high-risk patients, those with a dysfunctional form of TAS2R38, and improve approaches…

Delivering nutrients through dialysis could help people with co-occurring cystic fibrosis (CF) and kidney disease meet their nutritional requirements, a recent case report illustrates. The report, “Intradialytic parenteral nutrition improves nutritional status in a complex cystic fibrosis patient with redo double lung transplant and end-stage renal…

Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized triple combination for cystic fibrosis (CF) patients without available treatment options, from the Dutch Medicines Evaluation Board. Regulatory officials outlined necessary steps in this planned, pivotal trial that would support…