cystic fibrosis

People with cystic fibrosis (CF) infected with the new coronavirus appear to have better outcomes than initially anticipated, with their COVID-19 disease following a course similar to that of the general population, an international study suggests. This research also points to a lower incidence of coronavirus infections in…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for a label extension of the cystic fibrosis (CF) treatment Kalydeco (ivacaftor). If approved by the European Commission, the extension would allow Kalydeco use in patients 6…

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…

Orkambi (ivacaftor/lumacaftor) can improve glucose tolerance in people with cystic fibrosis, a small study in patients either at risk of or newly diagnosed with diabetes suggests. The study, “Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients,” was published in 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 …

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…

Zikani Therapeutics has raised $7.5 million in Series A funding to advance work, using its TURBO-ZM technology platform, into potential therapies for rare disorders caused by nonsense mutations, including cystic fibrosis (CF). TURBO-ZM is a proprietary platform that enables Zikani to rapidly produce ribosome modulating agents…

Delpharm has agreed to manufacture AzurRx BioPharma‘s investigational therapy MS1819 — a potential treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF) in clinical testing— using enteric capsules, an approach that increases the delivery of lipases (digestive enzymes). Delpharm produced a first batch of…