European researchers have identified a group of genes that behave as potential modifiers in cystic fibrosis (CF) patients with a F508del mutation in the CFTR gene — a discovery that may lead to new ways to treat the disease. Their study, “Transcriptomic profile of cystic fibrosis patients identifies type I…
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Portland Book Review (PBR) has given four stars to a new book, “There Are No Alligators in Heaven!,” that describes the hardships of a family struggling with cystic fibrosis (CF). The book is centered around Jennifer Hale, who was diagnosed with CF at the age of 2 but lived to…
Chest X-rays commonly used to monitor cystic fibrosis (CF) patients expose these people to cumulative radiation, a known risk for cancer. A study by researchers in Britain, published in the journal BMJ Open, found that newer equipment lessened this exposure and risk but levels were still a concern, especially for young CF patients…
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…
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