cystic fibrosis

NetNoggin has launched a new cystic fibrosis (CF) market research report to highlight patient and caregiver experiences and their perspectives about the gaps in CF research, the company announced. NetNoggin is a company that specializes in pharmaceutical marketing. The new offering, called NetNog: #genesis Cystic Fibrosis Patient and Caregiver…

Using lower, or suboptimal, doses of antibiotics to treat lung infections in children and young adults with cystic fibrosis (CF) leads to fewer changes in airway bacterial diversity compared to therapeutic (higher) antibiotic exposure. The findings also suggested, however, that patients receiving therapeutic doses had greater improvements in lung…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Despite being commonly found in patients with cystic fibrosis-related diabetes, the bacteria Stenotrophomonas maltophilia cannot be considered as an independent risk factor for developing CFRD, study says. The study, “Cystic fibrosis related diabetes is not independently associated with increased Stenotrophomonas maltophilia infection: Longitudinal data from the UK CF Registry,”…

A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases, including cystic fibrosis (CF), a study reports. This newly developed approach uses man-made RNA molecules, called anticodon engineered transfer RNAs or ACE-tRNA, that prevent the detection of abnormal stop signals in…

Researchers at Case Western Reserve University School of Medicine have received a $3.34 million grant from the National Institutes of Health (NIH) to develop a new strategy to sensitize Burkholderia multivorans — a common pathogen in patients with lung diseases like cystic fibrosis (CF) — to the action of…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Joint disease, or arthropathy, is common among people with cystic fibrosis (CF), and more likely to occur in older and female patients, and those who have more pulmonary flares or elevated blood levels of IgG antibodies, a study suggests. The higher number of flares and increased…

Exposing cystic fibrosis (CF) patients to high levels of oxygen through hyperbaric oxygen treatment may make Pseudomonas aeruginosa bacterial aggregates more sensitive to antibiotics like tobramycin and easier to eradicate from the lungs, a study in patient-derived bacterial cultures reports. The study, “Hyperbaric oxygen treatment increases killing of aggregating Pseudomonas aeruginosa isolates from…

Results from a Phase 3 trial demonstrate that Symkevi (tezacaftor and ivacaftor combo), known as Symdeko in the U.S., is safe and can effectively improve lung clearance in children, from 6 to 11 years old, with cystic fibrosis (CF). With these results, Vertex Pharmaceuticals — the…