PTC Therapeutics’ ataluren has failed to significantly improve respiratory function in nonsense mutation cystic fibrosis (nmCF), a severe form of the disease, according to results of a Phase 3 clinical trial. “We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause…
News
Sizable doses of a powerful class of antibiotics used to treat cystic fibrosis (CF) can accumulate over time to increase the risk of permanent hearing loss, according to a study. Previous research had shown an association between aminoglycosides and hearing loss. The new study is the first to address both daily…
Researchers conducting a Phase 1 study for cystic fibrosis (CF) have dosed their first healthy volunteer with a combination of the novel drugs GLPG2222 (a corrector) and GLPG2451 (a potentiator), the Belgian company Galapagos announced. The randomized, placebo-controlled study (NCT02788721) involves at least 40 healthy participants in Belgium.
Spyryx Biosciences has marked Feb. 28, Rare Disease Day, by announcing it will initiate a Phase 1b study to assess the safety of its SPX-101 in cystic fibrosis (CF) patients. SPX-101 is an experimental peptide designed to block the function of the lungs’ sodium channels, which move sodium and water from the…
The investigative oral treatment Resunab (JBT-101) shows promise in easing inflammation mediated by immune cells in the lungs of cystic fibrosis (CF) patients, according to results of a preclinical study. Results from a Phase 2 study of the treatment’s safety and efficacy in patients are expected shortly. The preclinical findings will be presented at Research and Development…
Kalydeco (ivacaftor) is known to increase the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in cystic fibrosis (CF) patients. A new study says the drug also improves lung function and reduces inflammation, but does not eradicate Pseudomonas bacteria infection. The study, “Restoring CFTR Function Reduces Airway Bacteria…
Bone marrow cells can improve overall lung function and may have potential therapeutic benefits in treating cystic fibrosis, suggests a study published in the journal Molecular Therapy.
Anti-infective agents such as Mycamine and Coly-mycin M can help prevent formation of Exophiala dermatitidis associated-biofilm structures, commonly found in cystic fibrosis (CF) patients, a new study found. The study, “Biofilm formation of the black yeast-like fungus Exophiala dermatitidis and its susceptibility to antiinfective agents,” appeared in…
Researchers found a new defect called the R248G mutation in the CFTR gene in three Spanish siblings whose diagnoses indicate borderline cystic fibrosis, and suggest a possible link between this defect, pancreatitis (inflammation of the pancreas), and infertility. The findings were reported in the article “R248G cystic…
Scientists at the University of California-Riverside have developed a novel way to help target therapeutics for cystic fibrosis (CF) and other diseases. Their study, “Inhibition of nonsense-mediated RNA decay by ER stress,” appeared in the journal RNA. The information encoded in human DNA is first converted to RNA, and…
Your CF Community
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.