News

The U.S. Food and Drug Administration (FDA) has granted its orphan drug designation to ARV-1801 (sodium fusidate tablets), a treatment developed by Arrevus for patients with cystic fibrosis (CF). CF patients frequently develop lung infections due to the accumulation of thick mucus in their lungs, creating…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

More than 70% of home nebulizers used by children with cystic fibrosis (CF) are contaminated with microorganisms, posing a serious potential health risk to patients and underscoring a significant need for educational programs focused on nebulizer maintenance and hygiene, a research study found. The study also found a…

The Cystic Fibrosis Foundation has reached an agreement with Synspira Therapeutics to support the further development of SNSP003, an enzyme replacement therapy (ERT) designed to treat malabsorption syndromes, including those affecting patients with cystic fibrosis (CF). Malabsorption syndromes are a group of disorders that develop when there…

A new non-invasive tool, called XV technology, allows professionals to visualize airflow in a living lungs and could help in the diagnosis, monitoring, and treatment of cystic fibrosis (CF) and other respiratory lung diseases, scientists report. The technology was developed by researchers at Monash University, and has been patented by the…

Iron deficiency is common among adults with stable cystic fibrosis (CF) and is significantly associated with anemia, vitamin A deficiency, antacids use, and moderate to severe lung disease, a study shows. Prevalence rates of iron deficiency were found to vary widely due to poor agreement between…

The presence of heme B — a component of red blood cells — in the lungs is linked to worse lung function in people with cystic fibrosis (CF). That finding was described in the study “Refinement…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

People with cystic fibrosis (CF) who were treated with Orkambi (lumacaftor/ivacaftor) have significantly less accumulation of fat in the liver — a condition known as hepatic steatosis — which is often associated with CF, a study showed.  While hepatic steatosis is common in those…

Girls with cystic fibrosis (CF) under the age of six have higher levels of a tiny RNA molecule, miR-885-5p, in their blood compared with boys of the same age, which could be linked to the worse clinical outcomes normally seen in women with CF, a study suggests. The study,…