cystic fibrosis

The most influential cystic fibrosis advocacy groups in the United States and Europe have endorsed the idea of a Phase 1 clinical trial to test a triple-combo therapy that Proteostasis Therapeutics has developed, the company announced. Proteostatis said the endorsements came from the Cystic Fibrosis Foundation’s and European CF Society’s clinical…

Galapagos is advancing its development of a range of cystic fibrosis (CF) treatments intended for use in combinations — either with already approved CF therapies or with its own compounds — the Belgian biotech company announced. Phase 2 trials Among the advances is completion of a Phase 2 trial…

Researchers in Canada found new blood biomarkers that can more accurately detect cystic fibrosis (CF) in newborns than standard screening methods — a discovery that could lead to improvements in early identification of this disease. The study, “Metabolic Signatures of Cystic Fibrosis Identified in Dried Blood Spots for Newborn Screening Without…

Synspira received an award of up to $3 million from the Cystic Fibrosis Foundation to advance the clinical development of its lead candidate, SNSP113, for the treatment of pulmonary complications in cystic fibrosis (CF). Synspira is a company developing a…

A number of cystic fibrosis discoveries, therapy advances and other developments were reported by Cystic Fibrosis News Today during 2017. Now that the year is over, it’s time to review the articles that appealed most to our readers. Here are the 10 most-read articles of 2017, with a brief description of what…

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…

The overall rate at which women with cystic fibrosis are becoming pregnant dropped slightly in recent years — coinciding with the introduction of CFTR modulators and the clinical trials that led to their approval as CF therapies — but appears to be rising again to pre-trial levels, a study reports.

Pseudomonas aeruginosa is a bacteria that commonly infects cystic fibrosis patients. Now, researchers have designed a synthetic peptide that blocks the ability of these bacteria to cause disease. This discovery might be crucial in fighting infectious diseases and preventing bacteria from becoming resistant to nearly all the antibiotic drug options available.

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…