The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to ETD001, an investigational therapy aiming to improve mucus clearance in people with cystic fibrosis (CF). The designation is awarded by the FDA to aid the development of treatments for serious or life-threatening rare diseases —…
News
Mucus-producing cells in people with cystic fibrosis (CF) have lower levels of ST3Gal1, an enzyme that sugarcoats mucus to make it less thick and sticky, but treatment with Trikafta can partly restore the enzyme and make mucus easier to clear away. The findings, from a study led by…
Non-white Canadians with cystic fibrosis (CF) have a higher risk of death than white Canadians with the disease, according to a study of nearly 6,000 people with CF. Despite having similar characteristics at diagnosis and similar rates of CF complications, non-white CF patients also had worse lung function,…
In laboratory experiments, a team of researchers in Belgium identified a few strategies that might help prevent bacteria from forming treatment-resistant clusters called biofilms that are believed to underlie chronic rhinosinusitis (CRS) — an inflammatory condition affecting the nasal passages and…
Detecting Nocardia bacteria doesn’t necessarily indicate therapy is needed for people with cystic fibrosis (CF), but an active infection should be closely monitored and patients with lung involvement, a decrease in lung function, or who are undergoing a pulmonary transplant should be treated, a review study suggests.
Long-term treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) led to significant gains in lung function and nutritional status for cystic fibrosis (CF) patients with advanced lung disease, a real-world analysis shows. The improvements weren’t as large as those seen in patients with milder lung disease, but the findings demonstrate the…
Lung function decline slowed consistently over five years with Kalydeco (ivacaftor) treatment in adults and children with cystic fibrosis (CF), a real-world study in the U.S. shows. “Overall, these results expand on those from prior studies and show that [Kalydeco] has a meaningful and sustained impact on reducing…
The Cystic Fibrosis Foundation has awarded $3 million to support a study of mental health among young children with cystic fibrosis (CF) to University at Buffalo (UB) researcher Beth A. Smith, MD, a psychiatrist whose work in this area has already helped to get new screening programs into…
About half of cystic fibrosis (CF) patients had used, were using, or were interested in using cannabidiol (CBD), the main active compounds found in the cannabis plant, according to a new survey and analysis conducted at a U.S. center. “Cannabidiol is used to treat a variety of symptoms including…
More severe lung disease and using inhaled corticosteroids are associated with shorter telomere length, a sign of cellular aging, in immune cells from people with cystic fibrosis (CF), a study suggests. Telomeres are stretches of repetitive DNA that protect the ends of chromosomes, the long strings of DNA that…
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