Marisa Wexler, MS,  senior science writer—

Treatment with the CFTR modulator Kalydeco (ivacaftor) led to significant weight gain, suggesting better nutritional status, in  babies and toddlers with cystic fibrosis (CF) eligible for this therapy’s use, a study reports. “Young children experienced favorable changes in weight gain and some nutritional biomarkers in the first 12…

Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) can improve lung function and nutritional status for people with cystic fibrosis (CF) caused by the N1303K mutation, a small real-world study reports. The N1303K mutation is not among the 178 disease-causing mutations in the CFTR gene considered to be responsive to Trikafta and…

Researchers have synthesized two new molecules that are able to kill Staphylococcus aureus and Pseudomonas aeruginosa — two species of bacteria that commonly cause lung infections in people with cystic fibrosis (CF). These new molecules, known as synthetic nano-engineered antimicrobial polymers or SNAPs, showed synergistic bacteria-killing effects when combined with…

Dosing has begun in a Phase 1 clinical trial of CM001, an inhaled treatment designed to act as a molecular prosthetic in cells and treat cystic fibrosis (CF) regardless of a patient’s underlying mutation, Cystetic Medicines, the therapy’s developer, announced. Most immediately, the company expects that CM001…

In the months after starting on Trikafta, people with cystic fibrosis (CF) generally experience lower rates of hospitalizations and lung infections — but nonetheless, the total cost of healthcare for these patients tends to increase, a new economic analysis indicates. The findings are in line with prior analyses…

Top-line results are expected next month for the SPAN clinical trial, a Phase 2 study testing First Wave BioPharma‘s new formulation of adrulipase to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). The trial’s target enrollment has been reached, with 13 total participants signed on,…

Long-term treatment with Trikafta leads to sustained improvements in lung function, easing of respiratory symptoms, and better nutritional status for people with cystic fibrosis (CF) who have at least one copy of the F508del mutation. That’s according to new data presented by the therapy’s developer Vertex Pharmaceuticals…

Rates of screening for diabetes among people with cystic fibrosis (CF) are not where they should be, a new study reports. “Studies to improve our approach to [CF-related diabetes] screening are urgently needed,” the researchers wrote in “A provider survey of cystic fibrosis related diabetes screening and…

For people with cystic fibrosis (CF), key barriers to getting specialty care include concerns about addiction and affordability, according to a new survey of clinicians. In fact, more than one-third of respondents cited difficulties for CF patients in accessing specialists and issues with insurance and treatment costs. “In this…

For people with cystic fibrosis (CF) taking Pulmozyme (dornase alfa), adding treatment with hypertonic saline — or HS for short — did not significantly improve lung function or reduce the risk of exacerbations compared with the approved therapy alone, a new study indicates. The findings showed that…