An Australian scientist has received a $1 million Cystic Fibrosis Foundation grant to study whether probiotics can improve the health and life expectancy of children with the disease. Keith Ooi, a University of New South Wales pediatric gastroenterologist, will look at whether daily probiotics can improve the gut bacteria mix and…
Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) approved Symdeko (tezacaftor/ivacaftor and ivacaftor) as a therapy for cystic fibrosis (CF) patients carrying two copies of the F508del mutation in the CFTR gene (the gene defective in CF) or with one mutation that responds to tezacaftor/ivacaftor. The therapy was approved for…
Savara Pharmaceuticals announced the publication of two case reports demonstrating that inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) either eradicated or dramatically reduced Mycobacterium abscessus (M. abscessus) infection, improved clinical outcomes, and was well-tolerated in patients with cystic fibrosis (CF). The results were published in the European Respiratory Journal in an…
The U.S. Food and Drug Administration (FDA) gave a green light to human testing of ORC-13661,  Oricula Therapeutics‘ treatment candidate to prevent hearing loss in people taking high doses of aminoglycoside antibiotics, including patients with cystic fibrosis. Approval given the company’s Investigational New Drug Application will enable Oricula to test…
Anthera Pharmaceuticals has completed the last patient visit in the primary treatment phase of a Phase 3 clinical trial evaluating Sollpura (lipromatase) for exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). Two interim analyses conducted by an independent data monitoring committee appointed by the Cystic…
The interaction of ions and sugars called glycans can affect the mucus that lines airways — a finding that has implications for cystic fibrosis, two studies reported. CF is characterized by abnormally thick and sticky mucus that can damage the lungs, gut and other organs and breed bacteria. The two studies showed…
Celtaxsys has obtained four U.S. patents, including a new one for acebilustat, its lead candidate for treating the lung inflammation found in cystic fibrosis. The other three patents cover potential therapies for additional inflammatory diseases. Acebilustat is designed to reduce an overabundance of white blood cells known as neutrophils in CF…
The sweat test, a clinical standard for diagnosing cystic fibrosis (CF), was found to be equally reliable for diagnosing CF in Down syndrome patients, according to a study published in the journal BMC Pulmonary Medicine. Previous case reports have suggested an association between CF and Down syndrome. Although rare, the…
Vertex Pharmaceuticals will advance to the next clinical-trial stage two therapies designed to correct faulty mechanisms underlying cystic fibrosis. The company said it plans Phase 3 trials of its next-generation correctors VX-659 and VX-445. They will be part of two triple combo therapy regimens that Vertex tests in CF patients. The…
A single dose of inhaled hypertonic saline (HS) solution increases mucus clearance for at least four hours in adults with cystic fibrosis (CF), concludes researchers at the University of North Carolina and John Hopkins University. Their study, “Hypertonic saline has a prolonged effect on mucociliary clearance in adults with cystic…
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