News

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 lowered the need for antibiotics and mucus-clearing therapies among children and adolescents with cystic fibrosis being followed at a pediatric CF center in Utah.  Jeffery T. Zobell, a clinical pharmacist at Primary…

Treatment with Trikafta rapidly eases symptoms of chronic nose and sinus inflammation in people with cystic fibrosis (CF), according to a new study — in which researchers noted that 30-60% of CF patients report symptoms of sinus disease. …

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…

The Cystic Fibrosis Foundation (CFF) is partnering with Pioneering Medicines, an initiative of Flagship Pioneering, to support the development of various types of gene therapy for cystic fibrosis (CF). The foundation is ready to invest up to $110 million to move therapy candidates into clinical testing, starting with an…

People with cystic fibrosis (CF) who start on the triple therapy Trikafta may be at greater risk of problems associated with excessive weight or obesity, such as metabolic syndrome — a group of disorders known to raise a risk of heart, liver, and kidney disease. “With the approval…