News

A potential gene therapy for cystic fibrosis, KB407, is able to induce the expression of normal CFTR protein in cell models of CF, its developer, Krystal Biotech, reports. These data are in the poster, “In vitro Pharmacology of KB407, an HSV-1 based gene therapy vector,…

A man-made antibiotic able to fight drug-resistant bacteria in the lungs with minimal toxicity to a person’s cells may be a promising approach to eliminate lung infections, such as those caused by resistant Pseudomonas aeruginosa, that trouble people with cystic fibrosis (CF), a study suggests. The antibiotic molecule…

The Cystic Fibrosis Foundation (CFF) is sponsoring free access to Beam, an online exercise platform that provides a variety of live and on-demand classes tailored for people with cystic fibrosis (CF), through Labor Day, Sept. 7. Complimentary registration through Beam’s website is available to adults with CF in the…

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…

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…