Hubrecht Organoid Technology (HUB) and the Dutch health insurance companies CZ, Zilveren Kruis and Menzis plan to launch a €3 million ($3.1 million) validation trial to determine whether HUB’s organoid technology can be used to assess how cystic fibrosis (CF) patients might respond to new therapies. HUB’s technology is based…
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Catabasis Pharmaceuticals’ compound CAT-5571 showed promising effects on CFTR activity (the defective gene/protein in cystic fibrosis) in preclinical models of cystic fibrosis when added to treatment with the drug Orkambi. Whether these results also hold true in humans will soon be answered, as Catabasis plans to launch a Phase…
Protalix BioTherapeutics’ AIR DNase (alidornase alfa), developed to make mucus in the lungs of cystic fibrosis (CF) patients less sticky, showed remarkably good results in an early analysis of a small, proof-of-concept Phase 2 trial (NCT02722122). The inhaled drug is a DNase, an enzyme that works to chop…
Corbus Pharmaceuticals has completed its Phase 2 clinical trial evaluating Resunab (JBT-101) for the treatment of adults with cystic fibrosis (CF), and expects to soon report topline results. “We look forward to having our first safety data on JBT-101 in CF,” Barbara White, MD, chief medical officer at Corbus,…
A number of important discoveries, therapeutic developments, and events related to cystic fibrosis (CF) were reported daily by Cystic Fibrosis News Today throughout 2016. Now that the year is over, it is time to briefly review the articles that appealed most to our readers. Here are the top 10 most-read…
Although patients with cystic fibrosis (CF) often have low levels of the antioxidant glutathione (GSH), the clinical benefits of GSH as an inhaled therapy appear to depend on the inflammatory status of their airways. The study, “Increasing sputum levels of gamma-glutamyltransferase may identify cystic fibrosis patients who do not…
Galapagos NV recently reported positive topline results from SAPHIRA 1, a Phase 2 clinical trial assessing the company’s potentiator molecule, GLPG1837, in patients with cystic fibrosis (CF) who have the G551D mutation. Data showed results comparable to an approved CF treatment, the company announced. SAPHIRA 1 (NCT02707562) was an…
Pregnant women with cystic fibrosis (CF) are more likely to develop diabetes and deliver prematurely by cesarean than other women, a retrospective study reports. They are also more likely to have babies with birth anomalies, although not necessarily life-altering ones. The study, “Maternal co-morbidities and neonatal outcomes associated with…
The bacteria Pseudomonas aeruginosa, which settle in the lungs of most cystic fibrosis (CF) patients, destroy the lungs by perpetuating the inflammation that aims to control invading microbes, according to a study. This contra-intuitive finding demonstrates the power of the mechanisms bacteria use to gain a survival advantage. It also…
In a small randomized clinical trial, researchers at Karolinska Institute in Sweden showed that vitamin D supplements may improve lung function in cystic fibrosis (CF) patients. A daily intake of high levels of vitamin D are required, however, for the effects to be noticed. The study, “Clinical…
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