The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to 4D-710, an inhaled gene therapy for people with cystic fibrosis (CF). The designation means 4D Molecular Therapeutics (4DMT) would be eligible to receive a priority review voucher from the FDA if its gene therapy…
The nasal microbiome — the community of microorganisms that live in the nose — obtained through nasal lavage correlates with inflammation in the nose and sinuses of children with cystic fibrosis (CF), a study indicates. The effect wasn’t observed when samples were obtained by a nasal…
No significant link was seen between an increase in body mass index (BMI), a measure of body fat based on weight and height, and higher blood fats and vitamin levels in people with cystic fibrosis (CF) being treated with Trikafta (elexacaftor/tezacaftor/ivacaftor), a study in adult and pediatric patients…
Discontinuing dornase alfa while on Trikafta (elexacaftor/tezacaftor/ivacaftor) could cut down on prescription costs by more than $1 billion each year in the U.S., according to estimates based on data from a randomized controlled trial. The savings would not compromise health, at least not for the short term, as people…
The diversity and relative abundance of microbes in the lungs — or the lung microbiome — was generally stable over an eight-month period in people with cystic fibrosis (CF), regardless of whether they had experienced a pulmonary exacerbation, according to a recent study. In the short term, using…
Two imaging techniques, called elastography and spectroscopy, can detect liver stiffness (hardness) and the buildup of fat in the liver in children with cystic fibrosis (CF) — particularly among those with CF-related liver disease — a study found. Importantly, because elastography and spectroscopy are noninvasive, these scans may offer…
Elevated levels of fecal calprotectin, measured over 1.5 years, significantly associated with poorer lung function in children with cystic fibrosis (CF), a European study reports. Levels of this protein, a biomarker of intestinal inflammation, also correlated with diarrhea in the young patients. Findings suggest that fecal calprotectin could originate…
The Cystic Fibrosis Foundation has honored the Pediatric Cystic Fibrosis Diagnosis and Treatment Center at Monroe Carell Jr. Children’s Hospital at Vanderbilt University with its 2023 Outstanding Care Center Partnership Award. The annual award is in recognition of the collaborations and commitment of the Nashville, Tennessee, center to…
The eighth annual BreatheCon, a virtual event hosted by the Cystic Fibrosis Foundation that brings together adults with cystic fibrosis (CF), is coming up Feb. 9-10. BreatheCon 2024 will focus on the individual experience of the CF journey. Participants will have opportunities to share their own stories,…
Results of three global clinical trials of a new triple-combination CFTR modulator therapy for cystic fibrosis (CF) —  vanzacaftor, tezacaftor and deutivacaftor — are expected shortly, Vertex Pharmaceuticals, the therapy’s developer, announced. Two of these Phase 3 trials, SKYLINE 103 (NCT05076149) and SKYLINE 102 (NCT05033080), which tested the…
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