Patricia Inácio, PhD,  science writer—

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…

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…

Volatile molecules present in bronchoalveolar lavage (BAL) samples from cystic fibrosis (CF) patients may help identify those infected with Pseudomonas aeruginosa and Staphylococcus aureus. The study, “Volatile molecules from bronchoalveolar lavage fluid can ‘rule-in’ Pseudomonas aeruginosa and ‘rule-out’ Staphylococcus aureus infections in cystic fibrosis patients,” appeared in the journal Scientific Reports. Pseudomonas aeruginosa is one…

AIT Therapeutics’ nitric oxide mist led to a 65 percent reduction in cystic fibrosis patients’ airway bacteria and an improvement in their exercise capability 60 days after treatment, a Phase 2 clinical trial shows. The bacteria that the nitric oxide countered was the Mycobacterium abscessus complex. The exercise-capability yardstick researchers…

Using a cystic fibrosis patient’s tissue to grow mini-guts in a lab has become easier and is   available to more scientists who want to use it both to study the disease and test patients’ response to therapies. Stemcell Technologies, a Canadian biotechnology company, has released two new lines…

When two channels that are supposed to move chloride and sodium ions out of cells in the lungs fail to function properly, it leads to the mucus buildup seen in cystic fibrosis. Japanese researchers have discovered that the channel dysfunctions also reduce the amount of zinc ions going into the…

British researchers have come up with a new way to screen for compounds that could be used to treat inherited diseases such as cystic fibrosis and Duchenne muscular dystrophy. The approach also may work with gene therapies, whose aim is to overcome abnormalities stemming from defective genes. These abnormalities can cause…

A pancreas abnormality is a sign that young children with cystic fibrosis may develop diabetes later in life, a study reports. The abnormality is fewer cells that produce and regulate blood sugar. These beta cells are part of clusters of different cells that scientists call islets. Researchers published their work…

Researchers determined the structure of a calcium-activated chloride channel, called TMEM16A, which they suggest could offer a new way of treating cystic fibrosis (CF). The study by scientists at University of Zurich, Switzerland, reporting these findings is titled “Activation mechanism of the calcium-activated chloride channel TMEM16A…

Canadian researchers developed a safer and more cost-effective method for transforming differentiated adult cells into stem-like cells, a process known as induced pluripotent stem cells (iPS). The new method, called interrupted reprogramming, already showed promise as a potential cell therapy replacement in a mouse model with cystic fibrosis. The study “…