cystic fibrosis

ProQR Therapeutics has finished a dose-escalating Phase 1b clinical trial evaluating the safety, stability and distribution throughout the body of its investigational therapy QR-010 in cystic fibrosis (CF) patients with a confirmed F508del CFTR gene mutation. Interim top-line results should be released by late September. A missing amino acid in the 508 position of the…

Air pollution increases the risk that young children with cystic fibrosis will develop the methicillin-resistant bacteria Staphylococcus aureus, or MRSA, a study reports. The research, “Air pollution exposure is associated with MRSA acquisition in young U.S. children with cystic fibrosis,” was published in the journal BMC Pulmonary Medicine.

AzurRx BioPharma has enrolled the sixth patient in a Phase 2 clinical trial of MS1819 as a treatment for a digestive disorder known as exocrine pancreatic insufficiency that is associated with cystic fibrosis. The company hopes to enroll 12-15 patients in the study (ACTRN12616000962437), which it is conducting in Australia and…

Good nutrition may slow the progression of cystic fibrosis patients’ lung disease, according to a review by researchers at the University of Oklahoma Health Sciences Center. It is especially important that cystic fibrosis patients with an end-stage lung disease not limit their consumption of healthy food, even if they…

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted for priority review applications for a combination of tezacaftor and Kalydeco (ivacaftor) as treatment for a subset of cystic fibrosis (CF) patients, Vertex Pharmaceuticals announced. “If approved, the tezacaftor/ivacaftor combination treatment would become Vertex’s third medicine to treat the underlying cause of…

Some children with severe infections who require intravenous antibiotics can be treated safely at home, according to a review of previously published studies. The review in the journal The Lancet Infectious Diseases reported that none of the studies found that home-based treatment was riskier than hospital care. The review also…

Inhalation of hypertonic saline solution (HSS) by children with cystic fibrosis (CF) increased sputum production and improved pathogen identification. These results suggest that this simple procedure, if implemented routinely in doctors’ offices and clinics, would help patients who can’t secrete mucus spontaneously. The study, “Hypertonic Saline as a Useful Tool…

Magnetic resonance imaging (MRI) scans, already important to both doctors treating patients and researchers conducting trials, might soon distinguish diseased and healthy tissue in differing colors, improving efforts to map and potentially diagnose and monitor diseases such as cystic fibrosis. A method allowing for two contrasting agents to be visible on MRI scans, called Dual…

Scientists have long known that defects in the CFTR protein cause cystic fibrosis. Now they’ve discovered that the defective protein leads to faulty insulin signaling, which, in turn, can cause diabetes and also contribute to cystic fibrosis complications. Insulin is a hormone that regulates metabolism, or the conversion of food to…

Corbus Pharmaceuticals plans to start a Phase 2b clinical trial of anabasum as a cystic fibrosis treatment by the end of 2017. The decision follows positive results of a Phase 2 study, the company said. Anabasum was formerly known as JBT-101 or Resunab. A Phase 2a trial usually looks at a drug’s effectiveness…