Vertex Pharmaceuticals selected its VX-445 (elexacaftor) triple regimen to apply for regulatory approvals in the U.S. and Europe over the third and fourth quarters of 2019. The therapeutic regimen is intended for the treatment of cystic fibrosis…
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Inhalation of nitric oxide as an add-on therapy may help reduce the burden of serious bacterial lung infections, namely the hard-to-treat Mycobacterium abscessus, in cystic fibrosis (CF) patients, AIT Therapeutics announced. The study with that finding, “Pilot study to test inhaled nitric oxide in cystic fibrosis…
International Biophysics, the maker of AffloVest, announced that it is continuing to support people with cystic fibrosis and efforts to raise awareness about this disease, donating 50 of its airway clearance therapy vests to be distributed across the U.S. AffloVests are being given away, one in each state,…
Treatment with ABBV-2222, an investigational CFTR corrector formerly known as GLPG2222, was well-tolerated and partially corrected the function of the CFTR protein, both alone and in combination with Kalydeco (ivacaftor), in a group of patients with cystic fibrosis (CF), Phase 2 clinical trials show. Trial findings…
AzurRx BioPharma has reached its patient enrollment goal in the ongoing Phase 2 trial testing the effectiveness of MS1819-SD, the company’s investigational therapy for the treatment of patients with exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF). MS1819-SD is a recombinant (artificial) form of an enzyme…
Long-term treatment with Kalydeco (ivacaftor) is well-tolerated and efficient, with improvements in sweat chloride concentration, growth, and pancreas function maintained over 84 weeks in 2-to-5-year-old children with cystic fibrosis (CF), results from the Phase 3 KLIMB extension study show. Trial findings were published in the study, “An…
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A mouse model carrying the most common mutation in the CFTR gene (the gene defective in patients with cystic fibrosis) shows disturbances in genes regulating the sleep cycle known as the circadian clock. The study “Dysregulation of Circadian Rhythm Gene Expression in Cystic Fibrosis Mice” was published in the…
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Expression of a peptide called RpoN* in antibiotic-resistant Pseudomonas aeruginosa isolates from cystic fibrosis (CF) patients made the bacteria more susceptible to antibiotics and reduced virulence in a roundworm infection model, a study has found. The study, “Blocking RpoN reduces virulence of Pseudomonas aeruginosa isolated from cystic…
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