Patricia Inácio, PhD, science writer —

New research from the University of Iowa’s Pappajohn Biomedical Institute (PBI) suggested that the sticky mucus found in cystic fibrosis (CF) patients is more viscous than usual because the thin layer coating the airways is more acidic in these people, a finding that supports a current therapeutic approach for CF. The study, “Acidic…

Ivacaftor (Kalydeco) treatment improves airway flexibility and capacity in patients with cystic fibrosis (CF), according to the results of a study, “Acute administration of ivacaftor to people with cystic fibrosis and a G551D-CFTR mutation reveals smooth muscle abnormalities,” published in the Journal of Clinical Investigation…

Arch Biopartners is collaborating with the University of Cincinnati (UC) for a new drug candidate, AB569, for treating Pseudomonas aeruginosa pulmonary infections in patients with cystic fibrosis. Last year, the U.S. FDA granted orphan drug status to Arch Biopartners’ AB569. The company also applied for orphan drug designation from the…

Young children with cystic fibrosis (CF) exhibit a more pro-inflammatory gastrointestinal microbiome, which may underlie impaired nutrient absorption, according to the study “Metagenomic evidence for taxonomic dysbiosis and functional imbalance in the gastrointestinal tracts of children with cystic fibrosis,” published in the journal Scientific…

A recent study describes a new assay, CFseq, that uses dried blood spots to screen for defects in the gene underlying cystic fibrosis (CF) in newborns, and allows for comprehensive, specific, and rapid early detection of the disease. The study, “Next-Generation Molecular Testing of Newborn Dried Blood Spots for Cystic…

Hepatopulmonary syndrome (HPS), a liver-induced lung disorder, may be underdiagnosed in at-risk patients with cystic fibrosis (CF), according to a study titled “Hepatopulmonary Syndrome in Patients With Cystic Fibrosis and Liver Disease,” published in the journal Chest. Cystic fibrosis-associated liver disease (CFLD) is the third cause of mortality among…

Researchers identified a novel, rare mutation causing cystic fibrosis (CF) in a pediatric African-American patient. The mutation, however, was found to be responsive to the CFTR corrector VX-809, therefore identifying potential personalized therapeutics. The study, “c.3623G > A mutation encodes a CFTR protein with impaired channel function,” was published…

Celtaxsys, a pharmaceutical company focused on novel therapeutics to treat inflammatory diseases, announced that it is beginning enrollment of patients for a phase 2 clinical trial to test the efficacy of acebilustat in patients with cystic fibrosis (CF). The study will be conducted in the United States and European Union…

In a recent study entitled “Comparative genomics of non-pseudomonal bacterial species colonising paediatric cystic fibrosis patients,” researchers performed a whole-genome profiling of bacterial strains, that are not Pseudomonas aeruginosa, from pediatric cystic fibrosis patients and determined their link to antibiotic resistance. The study was published in the open…

Concert Pharma recently announced successful results from a Phase 1 single ascending dose clinical trial for their lead investigational drug CTP-656 as a therapy for cystic fibrosis. Cystic fibrosis is a life-threatening disease characterized by a progressive lung function decline and is caused by mutations in the Cftr gene (cystic fibrosis transmembrane…