Steve Bryson, PhD,  science writer—

Evidence of immune dysfunction — elevated levels of immune cells and immune-related molecules — were found in the lungs of preschool children with cystic fibrosis (CF), even among kids without CF-related lung infections, a new study showed. Treatment with Kalydeco (ivacaftor), but not Orkambi (ivacaftor/lumacaftor), partially restored…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Porosome Therapeutics’ new treatment strategy for cystic fibrosis (CF), which is based on the porosome, a type of cellular structure. The designation is given to boost the development of therapies for rare diseases, which affect…

Nearly 80% of men with cystic fibrosis (CF) who underwent fertility treatment due to a lack of sperm became biological parents, according to the largest study of its kind. “Assisted reproductive technologies have removed many limitations to parenthood for CF males,” researchers wrote in the study “…

Prenatal Trikafta treatment of three fetuses with cystic fibrosis (CF) for an intestinal blockage called meconium ileus resulted in different outcomes, potentially due to treatment duration, according to a case series. One infant showed a complete resolution of meconium ileus at birth, one had persistent signs that were…

The U.S. Food and Drug Administration (FDA) has expanded the approval of Trikafta for people with cystic fibrosis (CF) ages 2 and older who carry certain mutations that are responsive to the medication. With the approval, 94 additional non-F508del CFTR mutations have been added to the medication’s label,…

Infants with cystic fibrosis (CF) have alterations to the nasal microbiome — the community of microbes that live in the nose — in the first year of life, a new study has discovered. These alterations, seen in newborns with CF but not in infants without the genetic disease, may…

About half of people with cystic fibrosis (CF) had breathing difficulties during sleep a year after a lung transplant, although the severity was mild, according to the first study to address the issue. Older age, being male, and having smaller static lung volume, which is when there is no…

Treatment with Kalydeco (ivacaftor) reduces the number of Pseudomonas aeruginosa bacteria in the lungs during an acute infection, but not during a chronic infection, a study using a rat model of cystic fibrosis (CF) indicates. Kalydeco led to better outcomes, including weight gain, increased CFTR protein function, and…

Researchers discovered a molecule that may help deliver gene therapies to cells in the thick mucus environment of the cystic fibrosis (CF) lung, a study reported. Since thick mucus makes gene delivery to the CF lung a significant challenge, “we are encouraged by these results and anticipate that our…

Since its approval in 2019, treatment with Trikafta has markedly reduced the number of lung transplants among people in the U.S. with cystic fibrosis (CF), with more than 35 patients removed from a transplant waitlist the year after the therapy was approved, according to a new analysis. Moreover,…