News

Stopping dornase alfa while on Trikafta may save $1B

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…

2 imaging techniques can detect liver issues in CF children: Study

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…

Fecal calprotectin levels tied to poorer lung function in children

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…

Vanderbilt’s pediatric center earns CF Foundation honor

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…

Registration is open for BreatheCon 2024, Feb. 9-10

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,…

Data awaited in Phase 3 trials of new triple-combo CFTR modulator

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’s eRNA may result in long-lasting CFTR production

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 raises $33M to develop non-antibiotic CF treatment

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…