Marisa Wexler, MS,  senior science writer—

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,…

Health Canada has approved Trikafta  as a triple-combination therapy for people with cystic fibrosis (CF), ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation. This decision is expected to give about 1,100 people access for a first time to a therapy that directly…

A new strategy to correct so-called splicing mutations could help deliver nucleic acid therapies called oligonucleotides to cells in the lung, a new study suggests. This approach may aid in the development of new treatments for people with cystic fibrosis (CF), according to researchers. “With our oligonucleotide delivery platform,…

A collaboration between the Cystic Fibrosis Foundation and Deep Science Ventures aims to identify technologies that might overcome the challenges inherent in developing gene therapies for cystic fibrosis (CF). The effort is part of the foundation’s Path to a Cure initiative, a $500 million research program whose focus…

BX004, a phage therapy candidate for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), was able to effectively kill a range of antibiotic-resistant bacterial strains under laboratory conditions, new data show. These findings were shared in the poster “Phage therapy for chronic Pseudomonas aeruginosa infections in Cystic Fibrosis…