A noninvasive measurement of lung function called lung clearance index (LCI) was associated with the degree of lung damage over time in children with cystic fibrosis (CF), according to a recent study. LCI could be used to monitor lung disease progression in children with CF and may help clinicians…
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KIT2014, an experimental treatment that Kither Biotech is developing to treat cystic fibrosis (CF) and other lung diseases, was able to reduce inflammation, relax the airways, and improve the functionality of CFTR modulators in preclinical models, a study shows. Kither is planning to launch a Phase 1/2a clinical…
In children with cystic fibrosis (CF) and a first or new-onset Pseudomonas aeruginosa infection, antibiotics given intravenously are not more effective at eliminating the bacteria than inhaled antibiotics given alone or as combination therapy, a Turkish study suggests. Intravenous (IV) treatment was also associated with more pulmonary exacerbations and poorer lung function after…
The Cystic Fibrosis Foundation (CFF) has awarded up to $15.9 million to Eloxx Pharmaceuticals to support the company’s clinical program for ELX-02, an investigational therapy for cystic fibrosis (CF) patients with nonsense mutations. A total of $7 million will be provided upfront, with additional funding granted based on…
Beginning April 1 the Australian government will subsidize the cost of Trikafta (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF) under the Australian Pharmaceutical Benefits Scheme (PBS), dramatically reducing out-of-pocket expenses for eligible Australians. The subsidy was made possible by a reimbursement agreement reached between Trikafta’s developer, Vertex…
Health Canada has approved Vertex Pharmaceuticals’ Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in patients 4 months to 18 years old who carry the R117H mutation in the CFTR gene, and who weigh at least five kilograms (about 11 pounds). “It’s been our goal to ensure that…
Levels of several metabolites were associated with inflammation and the presence of bacteria in the lower airways of people with cystic fibrosis (CF), which could be used as disease biomarkers, according to a recent study. “Our study was the first to both examine these metabolites in lower airway samples and…
More than 160 cystic fibrosis (CF) patients representing 46 states met virtually with congressional lawmakers to advocate for legislation that would provide a more robust antibiotics pipeline. As part of the Cystic Fibrosis Foundation’s (CFF) 15th annual March on the Hill, advocates sought sponsorship support for the…
Elevated levels of DMBT1 in the lungs, a protein previously linked with inflammatory processes, may be a biomarker of progression in cystic fibrosis (CF) patients, a study suggests. High DMBT1 levels impaired the movement of cilia, the finger-like projections that help to clear mucus from the airways, its scientists…
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
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