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…
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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…
Antibiotics are less effective at treating lung infections in cystic fibrosis (CF) when there are multiple types of infectious microorganisms present, according to a new study. “People with chronic infections often have co-infection with several pathogens, but the problem is we don’t take that into account in deciding how…
Co-infection with the bacterium Pseudomonas aeruginosa can increase the potency of therapies that kill Candida albicans, an infectious fungus, according to a new study. The study, “Pseudomonas Synergizes with Fluconazole against Candida during Treatment of Polymicrobial Infection,” was published in Infection and Immunity. Pseduomonas and Candida are both microorganisms…
Survival following lung transplant due to cystic fibrosis (CF) in children ages 10 and younger was similar to survival among older CF children with one key difference, a hospital study suggests. While recognizing and treating diabetes, infections, and other lung conditions early may prevent or slow lung disease progression,…
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