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…
News
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…
Sail Biomedicines’ experimental programmable RNA technology, called Endless RNA or eRNA, appears to result in long-lasting production of the protein that is missing or faulty in people with cystic fibrosis (CF), according to research funded by the CF Foundation. Producing the protein over an extended period of…
Clarametyx Biosciences has completed a $33 million financing round to support its development of CMTX-101, a non-antibiotic treatment candidate for persistent bacterial infections in people with cystic fibrosis (CF). The funding contributed to the start of a Phase 1b/2a clinical trial of CMTX-101 as an adjunct therapy to standard-of-care…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to BX004, BiomX’s inhaled phage therapy being developed for chronic Pseudomonas aeruginosa pulmonary infections in people with cystic fibrosis (CF). The designation seeks to encourage new therapies for rare diseases, those affecting fewer than 200,000…
Your CF Community
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.