First Wave BioPharma is teaming up with Rho for a Phase 2 clinical trial to test their new adrulipase formulation, designed as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). “We are eager to…
News
Twice-weekly sessions of airway clearance physiotherapy over eight weeks led to clinically meaningful reductions in respiratory symptoms in people with cystic fibrosis (CF) in a small clinical trial. These preliminary findings, based on patient-reported measures, support the need for further studies to determine the most effective airway clearance technique,…
Researchers in Oregon have developed lipid nanoparticles that can be inhaled and effectively deliver messenger ribonucleic acid (mRNA) into the lungs of mice, triggering the cells to produce the defective protein in cystic fibrosis (CF). “Inhaled [lipid nanoparticles] resulted in localized protein production in the mouse lung without toxicity,…
For a small group of people with cystic fibrosis (CF), one year of treatment with Orkambi (ivacaftor/lumacaftor) improved bone health and stabilized pancreatic function, nutritional status, reproductive hormone levels, and lung function. These findings from a recent study in Israel demonstrate the use of Orkambi, which targets the…
Treatment with Orkambi (lumacaftor/ivacaftor) for six months was found to improve exercise endurance in three adults with cystic fibrosis (CF), a new study showed. Moreover, by the end of the study, all three men experienced less leg discomfort and less dyspnea, or shortness of breath. For two patients,…
The incidence of colorectal cancer (CRC) in people with cystic fibrosis (CF) was five times higher than in those without CF, after adjusting for age, in a large-scale English population study. CFTR gene mutations, the underlying cause of CF, occurred more frequently than expected, suggesting an association between…
Prolonged use of certain medications used to treat acid reflux, called proton pump inhibitors (PPIs), may put people with cystic fibrosis (CF) at higher risk for Pseudomonas aeruginosa infection, according to a single-center study in Italy. Long-term treatment was also associated with more hospitalizations for pulmonary exacerbations, or episodes of…
Newborn genetic screening panels for variants in the CFTR gene — mutations of which are the underlying cause of cystic fibrosis (CF) — had lower detection rates in minority racial and ethnic groups in the U.S., a large-scale analysis found. Lower detection rates with genetic testing among these…
A recent overall decline in lung transplant rates for adults and children with cystic fibrosis (CF) in the U.S. has had less impact on nonwhite patients, according to a recent analysis. While transplant rates have decreased in the past couple years for all people with CF, the declines were…
Women with cystic fibrosis (CF) have different experiences with sexual and reproductive health than their peers without the disease, necessitating special considerations in their care, a new study shows. The study, “Sexual and reproductive health experiences and care of adult women with cystic fibrosis,” was published in the…
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