cystic fibrosis

AzurRx BioPharma has reached its patient enrollment goal in the ongoing Phase 2 trial testing the effectiveness of MS1819-SD, the company’s investigational therapy for the treatment of patients with exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF). MS1819-SD is a recombinant (artificial) form of an enzyme…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Long-term treatment with Kalydeco (ivacaftor) is well-tolerated and efficient, with improvements in sweat chloride concentration, growth, and pancreas function maintained over 84 weeks in 2-to-5-year-old children with cystic fibrosis (CF), results from the Phase 3 KLIMB extension study show. Trial findings were published in the study, “An…

A mouse model carrying the most common mutation in the CFTR gene (the gene defective in patients with cystic fibrosis) shows disturbances in genes regulating the sleep cycle known as the circadian clock. The study “Dysregulation of Circadian Rhythm Gene Expression in Cystic Fibrosis Mice” was published in the…

Expression of a peptide called RpoN* in antibiotic-resistant Pseudomonas aeruginosa isolates from cystic fibrosis (CF) patients made the bacteria more susceptible to antibiotics and reduced virulence in a roundworm infection model, a study has found. The study, “Blocking RpoN reduces virulence of Pseudomonas aeruginosa isolated from cystic…

The incidence rate of cystic fibrosis-related diabetes (CFRD) in children with cystic fibrosis (CF) has remained stable over time, according to a Canadian multi-center registry study. According to the researchers, these findings contrast with increasing incidence rates observed for type 1 and type 2 diabetes in the general…

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…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

When it comes to disease “awareness” days, weeks, and months, 2019 is crammed with hundreds of them. May leads the calendar with 37 such designations, calling attention to everything from stuttering and scleroderma to high blood pressure, lupus and skin cancer. Cystic Fibrosis Awareness Month also occurs in May. It’s…

Poor oxygen conditions, a common trait in the lungs of cystic fibrosis (CF) patients, may help Pseudomonas aeruginosa bacteria outcompete other infectious agents, namely Staphylococcus aureus, a study suggests. The study, “Anaerobiosis influences virulence properties of Pseudomonas aeruginosa cystic fibrosis isolates and the interaction with Staphylococcus aureus,” was…