Marisa Wexler, MS,  senior science writer—

The triple-combination therapy Trikafta significantly improved lung function and life quality in a global Phase 3 trial that enrolled people with cystic fibrosis (CF) caused by one F508del mutation, and one gating mutation or one residual function mutation, in the CFTR gene. Data from the…

Health Canada has extended the use of Kalydeco (ivacaftor) to treat babies, starting at 4 months old, who have cystic fibrosis (CF) caused by certain mutations. The specific gating mutations covered are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. Kalydeco, Vertex Pharmaceuticals, was previously approved in…

The Calgary and Southern Alberta Chapter of Cystic Fibrosis Canada has announced that this year’s 65 Roses Ladies Golf Classic, a women-only golf tournament to raise funds and awareness for cystic fibrosis (CF), will take place Sept. 13 at Earl Grey Golf Club in Calgary. The event offers…

Voting is open for the 2021 Thriving Undergraduate and Thriving Graduate Scholarship, a program run by AbbVie that awards scholarships totaling $25,000 to one undergraduate and one graduate student with cystic fibrosis (CF). AbbVie selected 40 undergraduate and graduate students living with CF earlier this year, with each…

The World Health Organization (WHO) proposed a compound name to better identify MS1819, an investigational therapy for exocrine pancreatic insufficiency in people with cystic fibrosis (CF), with scientists globally. MS1819 may soon be called “adrulipase alfa.” The WHO is expected to make a final decision on the candidate’s International Nonproprietary…

Combining investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI), top-line results from a Phase 2 clinical trial show. “A safe and effective therapy that allows CF patients to gain control over…

People with cystic fibrosis (CF) who use opioids to manage pain commonly report being satisfied with the treatment, but also have concerns about the potential for addiction and stigmatization, according to the results of a recent patient survey. The findings highlight “the need for guideline-driven practice standards, including the…

Among people with cystic fibrosis (CF), antibiotic-resistant Staphylococcus aureus is sometimes transmitted among relatives, but rarely in healthcare settings, an analysis of patients at a CF center in Iowa suggested. Findings also indicate that distinct subsets of S. aureus may be associated with differences in disease progression. These results were in the…

A novel compound that may hold promise for treating the roughly 11% of cases of cystic fibrosis (CF) that are caused by nonsense mutations has been described in a new study. The study, “A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1…

Eloxx Pharmaceuticals announced it expects to share data later this year from the first four treatment groups of a Phase 2 program evaluating the investigational therapy ELX-02 in people with cystic fibrosis (CF) who have at least one G542X mutation. “Given the substantial efforts of our clinical team,…