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,…
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A new triple-combination CFTR modulator therapy — vanzacaftor, tezacaftor, and deutivacaftor — has been approved by the U.S. Food and Drug Administration (FDA). The therapy, which developer Vertex Pharmaceuticals will market under the name Alyftrek, is indicated for cystic fibrosis (CF) in patients 6 and older who carry…
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…
Treatment with Trikafta may help ease severe symptoms of anxiety and depression in children with cystic fibrosis (CF), a study in the U.S. suggests. The study, “Mental Health of Pediatric Patients with Cystic Fibrosis after 18 Months on Elexacaftor-Tezacaftor-Ivacaftor Therapy,” was published in Pediatric Pulmonology. The study’s researchers…
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…
The season and climate influence the age and risk of the first bacterial exposure or infection, that is, initial acquisition, in cystic fibrosis (CF) patients, a study suggests. Methicillin-sensitive Staphylococcus aureus (MSSA) and Pseudomonas aeruginosa are the primary causes of infections in CF patients. A MSSA infection occurs earlier in life than…
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…
Children and adolescents with cystic fibrosis (CF) interviewed for a study expressed a desire for active participation in the hospital setting and during CT scans, and wanted greater involvement during longer, more painful procedures. “It’s a fair right to have, after all, [children and adolescents are] the ones [it]…
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,…
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