Alejandra Viviescas PhD,  —

Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days of treatment with intravenous antibiotics could help predict treatment response in cystic fibrosis patients experiencing pulmonary exacerbations, a study has found. The study, “Identification of novel blood biomarkers of treatment response…

AzurRx BioPharma has started dosing patients in a Phase 2 clinical trial assessing its investigational therapy MS1819-SD in combination with porcine enzyme replacement therapy (PERT) for people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI). People with CF can develop EPI when…

Viral respiratory tract infections in young children with cystic fibrosis tend to be mild and not require hospitalization, a study found. Infections caused by respiratory syncytial virus (RSV) were rare and did not lead to complications, suggesting that there is no need for preventive medication such as…

High blood levels of the enzyme alanine aminotransferase (ALT)  — a biomarker of liver damage — might indicate a higher risk of rising blood sugar levels and cystic fibrosis-related diabetes (CFRD), especially in men, a study found. These data suggest that ALT could serve as a marker of CFRD risk…

The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all types of mutations in the CFTR gene. The funding will establish a multi-year program aimed at boosting the development…

Kalydeco (ivacaftor) preserved lung function, improved the nutritional status, and reduced hospital visits and chronic bacterial infections in people with cystic fibrosis (CF) when administered for up to five years in a clinical setting, a long-term, real-world, observational study showed. The study, “Disease progression in patients…

The inhaled formulation of broad-spectrum antibiotics such as imipenem/cilastatin is a useful alternative to treat chronic lung bacterial infections in children with cystic fibrosis (CF) because it causes fewer side effects, two case reports suggest. The study, “Use of inhaled imipenem/cilastatin in pediatric patients with cystic…

A case report about a 10-year-old boy suggests that Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) can restore the function of the pancreas and the production of pancreatic enzymes in children with cystic fibrosis (CF), leading to a reduction or discontinuation of pancreatic enzyme replacement therapy (PERT). The…

Vivus recently announced a partnership with HarborPath Direct to facilitate access to Pancreaze (pancrelipase) for eligible patients who do not have health insurance or are underinsured. The treatment will be available through the Pancreaze Advantage Program, which is integrated with HarborPath’s patient assistance program. Patients can…

The case report of a cystic fibrosis (CF) patient suggests that phages — viruses that naturally infect and kill bacteria — are a potential personalized therapy against infections caused by antibiotic-resistant bacteria. The study, “Engineered bacteriophages for treatment of a patient with a disseminated drug-resistant Mycobacterium…