A single dose of 4D Molecular Therapeutics’ inhaled gene therapy candidate 4D-710 improved or stabilized lung function in adults with cystic fibrosis (CF), according to interim data from a Phase 1/2 clinical trial. Additional data from the AEROW (NCT05248230) study, which is evaluating the therapy in CF…
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Levels of fat-soluble vitamins D and E and of the micronutrients zinc and selenium are generally within the normal range in people with cystic fibrosis (CF), even during treatment with Kaftrio. That’s according to a recent study that also found significantly increased vitamin A levels one year into…
Extended treatment with Kaftrio progressively increases the diversity of the microorganisms in the gut in people with cystic fibrosis (CF), a recent study suggests. However, gut microbiota diversity remained significantly different from healthy people after about 1.5 years on the CFTR modulator, probably due to the use…
Women with cystic fibrosis (CF) who have been pregnant three or more times are more likely to experience a greater decline in lung function and, when 30 or older, to give birth to preterm babies or babies with complications, a study has found. Lung function appeared to decline more…
A miniaturized wearable device is able to measure chloride levels in sweat, the gold standard tool for diagnosing cystic fibrosis (CF). In infants with CF, tests showed the device’s accuracy matched standard methods without the need for trained personnel in hospital settings. It also measured sweat levels of zinc…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to SPL84, an experimental inhalation therapy for people with cystic fibrosis (CF) caused by the 3849+10 kb C-to-T mutation in the CFTR gene. The designation is given to accelerate the development and review of therapies meant to address unmet…
Two Massachusetts-based companies, Nanite and Saliogen Therapeutics, are teaming up on a new research project that aims to create a genetic medicine with the potential to treat cystic fibrosis (CF). “Nanite’s relationship with SalioGen is consequential in discovering safe polymeric nanoparticles for a transformative treatment for CF,”…
Certain neutrophils, a type of immune cell that’s released quickly into the bloodstream to help fight off infections, appear to be linked to better lung function in some people with cystic fibrosis (CF), a study finds. The presence or abundance in the blood of certain other immune cells and…
Exposure in utero to the cystic fibrosis (CF) medication Trikafta (elexacaftor/tezacaftor/ivacaftor) didn’t appear to negatively affect organ development in fetal rats whose mothers were given it, but it did change the activity of some genes, a study finds. While the functional impacts of the changes remain to be…
One year of treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor), branded Trikafta in the U.S., helped people with cystic fibrosis (CF) gain exercise capacity, particularly those who saw changes in weight and lung function, according to a study out of Denmark. While there was a significant increase in the highest amount…
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