Treatment with Orkambi (lumacaftor/ivacaftor) for six months lessened airway inflammation and enhanced airway bacterial diversity in people with cystic fibrosis (CF), but only when administered before patients were chronically infected with the bacterium Pseudomonas aeruginosa, a study has found. The findings suggest that “CFTR modulators [such as Orkambi] should…
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Lung function decline in adults with stable cystic fibrosis (CF) was tied to high numbers of mature low-density neutrophils, a white blood cell subtype associated with inflammatory disorders, a study suggests. While previous research showed that low-density neutrophils in CF children correlated with better lung function, the changes found…
The Cystic Fibrosis Foundation (CFF) is investing up to $15.5 million in Anagram Therapeutics to conduct early-stage trials of a new oral enzyme replacement therapy. The company is planning to start a Phase 1 clinical trial this summer. The therapy would let people with cystic fibrosis (CF)…
Pulmonary exacerbations in children with cystic fibrosis (CF) caused by multiple bacteria (polymicrobial) are generally treated with antibiotics that cover all the bacteria detected, a study reported. Whether this strategy is associated with better clinical outcomes than using antibiotics that cover only some of the detected bacteria remains to…
In a small clinical trial, Orkambi (lumacaftor/ivacaftor) appeared to reduce structural damage in the lungs, as measured by a chest MRI, for preschool-age children with cystic fibrosis (CF) who are homozygous for the F508del mutation. “This study suggests that [Orkambi] may modify CF disease progression when administered early…
Researchers are developing new inhalable nanoparticles designed to deliver RNA-based gene-editing therapeutics directly to the lungs, a study reports. Now shown to be effective in mice, the ultimate goal is to develop inhaled therapeutics to correct genetic defects in conditions affecting the lungs, such as cystic fibrosis (CF). “This…
A project that dissects the molecular proceses that allow bacteria of the Burkholderia cepacia complex to attach to cells was awarded a $400,125 grant from the National Institutes of Health (NIH). These bacteria are known to cause serious infections in people with cystic fibrosis (CF). The grant was awarded to Tung…
Nearly 39,000 people were estimated to be living with cystic fibrosis (CF) in the U.S. in 2020, up from the roughly 34,000 estimate made in 2012, a study led by the Cystic Fibrosis Foundation reported. A majority — about 77% — of these people also took part in…
American Airlines and its partners announced having raised $1.1 million for the Cystic Fibrosis Foundation (CFF) during the annual American Airlines Celebrity Ski event. The event, running for 38 years, has collected more than $45 million in support of the foundation’s goal of a cure for cystic fibrosis (CF). Last year’s…
Small changes in the shape of CFTR, the protein defective or absent in people with cystic fibrosis (CF), opened its gate outside the cell to allow the flow of chloride ions, a study reported. CF-causing mutations that suppress CFTR function disrupt these long-distance shape changes. By comparison, CFTR potentiator…
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