Marisa Wexler, MS,  senior science writer—

Children with cystic fibrosis (CF) who are older, thinner, or have worse lung function may be at increased risk of malnutrition, a study indicates. These findings highlight the importance of screening for malnutrition among children with CF. The study, “Triage for Malnutrition Risk among Pediatric and…

BiomX has unveiled a platform designed for more rapid and efficient development of phage therapy, which the company is using to test potential treatments, including one for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). According to a press release, the company expects results of a proof-of-concept…

A clinical trial testing AzurRx BioPharma‘s investigational therapy MS1819, in combination with the current standard care for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), has dosed its first two participants, AzurRx announced. “In the midst of a global pandemic…

The Cystic Fibrosis Foundation (CFF) has awarded up to $3.3 million to Polyphor AG to advance the testing of an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). According to a CFF press…

AzurRx BioPharma is requesting a new patient arm be added to its ongoing Phase 2b trial investigating MS1819 capsules as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). The request, filed with the U.S. Food and Drug Administration (FDA), would add a patient group…

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 National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

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…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of reducing pulmonary exacerbations, the investigational therapy’s developer, Corbus Pharmaceuticals, announced. “We are very disappointed that the study did not meet the primary endpoint,” Barbara White, MD, chief medical officer and…