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Trikafta treatment can reduce the frequency of pulmonary exacerbations — times when lung symptoms get markedly worse — for people with cystic fibrosis (CF). That’s according to an analysis of real-world data shared at this year’s North American Cystic Fibrosis Conference (NACFC), in an oral presentation, “…

Personalized treatment with bacteria-infecting viruses, called bacteriophages, could be used to combat Pseudomonas aeruginosa, the main bacteria involved in lung infections in people with cystic fibrosis (CF), both by killing the bacteria and making them less resistant to antibiotics. Bacteriophage therapy “is safe, decreases P. aeruginosa titers [levels], and…

Treatment with Trikafta may improve nutritional status or lessen anemia among some people with cystic fibrosis (CF) who have received a lung transplant, but more research is needed to understand the potential benefits — and risks — of using the triple-combination medication in this particular patient population. That’s…

Treatment with Trikafta improves lung function in people with cystic fibrosis (CF) who are not eligible for treatment with other CFTR modulators, according to interim data from an observational study. Results were shared at this year’s North American Cystic Fibrosis Conference (NACFC) in the presentation, “…

Clinicians who are caring for people with cystic fibrosis (CF) should talk to their patients about pregnancy and family planning “early and often,” according to researchers. In a presentation at the 2021 North American Cystic Fibrosis Conference (NACFC), Kristina Montemayor, MD, an assistant professor at Johns Hopkins University, also…

Six months of treatment with Trikafta does not substantially alter digestive symptoms in people with cystic fibrosis (CF), new research shows. “We speculate that [Trikafta], while it may lead to improvement in lung function, may have little or very delayed impact on gastrointestinal symptoms in people with CF,” Sarah…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

For children with cystic fibrosis (CF), newborn screening is associated with markedly improved real-world outcomes, including earlier diagnosis, greater weight and height gains, and fewer bacterial infections, new research demonstrates. In fact, infants are now diagnosed with the genetic disease at a median of 0 months…