Steve Bryson, PhD,  science writer—

A Chicago children’s hospital is leading an awareness campaign to reduce the missed or delayed diagnosis of cystic fibrosis (CF) after newborn screening, especially in non-white infants. “Delays in diagnosis of cystic fibrosis increase the risk for severe illness,” Susanna McColley, MD, principal investigator and a pulmonologist at the…

One year of treatment with hypertonic saline, inhaled twice daily, reduced the thickness of the airway walls in children with cystic fibrosis (CF), according to an analysis of clinical trial data. Researchers used an automated method to assess changes in lung structure captured on CT scans, and supported…

People with cystic fibrosis (CF) who were underweight before treatment gained the most weight after six months on Trikafta (elexacaftor/tezacaftor/ivacaftor), according to a new study. Underweight participants, as assessed with the body mass index (BMI), also saw the biggest gains in treatment-related lung function. Patients who were overweight…

Trikafta treatment for six months in people with cystic fibrosis (CF) was found in a real-world study to reduce the thickness of the airway walls and lead to less excessive mucus plugging in the lungs. Overall, as observed by MRI, the approved CF therapy improved structural abnormalities in…

A1c, a test that measures the average levels of sugar in the bloodstream, was able to predict the future risk of adult and pediatric cystic fibrosis-related diabetes (CFRD), a study shows. Elevated CFRD risk was associated with an A1c value of 5.5% or above in children and 6% and…

Researchers have identified a new class of CFTR correctors to treat cystic fibrosis (CF) that enhance the potency of currently available CFTR modulator therapies, a study shows. Data reveal that these compounds demonstrated high potency in airway cells derived from CF patients. The study, “Novel tricyclic…

A single dose of 4D Molecular Therapeutics’ 4D-710, an inhaled gene therapy candidate for adults with cystic fibrosis (CF), improved or stabilized lung function in three patients in a Phase 1/2 clinical trial. Nine to 12 months of follow-up data showed it led to meaningful gains in quality…

People with cystic fibrosis (CF) who frequently use healthcare resources accounted for almost one-half of total healthcare costs, according to a new Canadian study. Becoming a frequent high-cost CF healthcare user was linked to severe lung impairment, lung transplant, and liver cirrhosis (a chronic liver disease) with high blood…

Beyond improving lung function, Orkambi (ivacaftor/lumacaftor) has potent anti-inflammatory benefits that may limit immune-related lung damage in people with cystic fibrosis (CF), according to a real-world study. Researchers found that one year of treatment significantly reduced the levels of pro-inflammatory signaling molecules in the bloodstream and airways of…

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for children with cystic fibrosis (CF) as young as one month old, who have at least one gating mutation in their CFTR gene that is responsive to the oral treatment. The agency’s approval was supported by data…