Steve Bryson, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) will allow children with cystic fibrosis (CF) as young as age 2 to use Relizorb, an enzyme cartridge that connects with an enteral feeding tube to provide easier digestion of fats. “This additional RELiZORB clearance represents access for approximately 25% of…

Children and adolescents who are wait-listed for lung transplantation and have the worst functional status are at the greatest risk of being removed from the waitlist due to clinical deterioration or death, a study reports. The risk is highest for patients with cystic fibrosis (CF) and for adolescents compared…

CFTR modulator therapies can enhance the ability of certain immune cells to kill bacteria, independent of defects in the CFTR protein that cause cystic fibrosis (CF), according to a new study. Specifically, these modulators alter metabolic processes to activate anti-bacterial processes within both CF and non-CF immune cells known…

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…