Kalydeco (ivacaftor) preserved lung function, improved the nutritional status, and reduced hospital visits and chronic bacterial infections in people with cystic fibrosis (CF) when administered for up to five years in a clinical setting, a long-term, real-world, observational study showed. The study, “Disease progression in patients…
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The experimental treatment eluforsen is safe for adults with cystic fibrosis (CF) caused by a mutation called F508del, a new study reports, supporting further investigation and development of the treatment. The study, titled “Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic…
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EffRx Pharmaceuticals announced that it has signed an exclusive license agreement with Pharmaxis to register and commercialize Bronchitol (mannitol) for the treatment of cystic fibrosis (CF) in Switzerland. According to EffRx, Bronchitol should be available to Swiss patients by 2021. Bronchitol is a dry powder for…
Murepavadin (POL7080), Polyphor‘s lead investigational antibiotic targeting Gram-negative bacteria, behaved as expected and was well-tolerated when administered to mice harboring a lung infection caused by Pseudomonas aeruginosa, data from a preclinical study show. Of note, P. aeruginosa is a bacteria that often infects the lungs of patients…
The inhaled formulation of broad-spectrum antibiotics such as imipenem/cilastatin is a useful alternative to treat chronic lung bacterial infections in children with cystic fibrosis (CF) because it causes fewer side effects, two case reports suggest. The study, “Use of inhaled imipenem/cilastatin in pediatric patients with cystic…
A case report about a 10-year-old boy suggests that Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) can restore the function of the pancreas and the production of pancreatic enzymes in children with cystic fibrosis (CF), leading to a reduction or discontinuation of pancreatic enzyme replacement therapy (PERT). The…
Data from Phase 1 studies in healthy people and preclinical work indicate that ELX-02 may help to treat cystic fibrosis (CF), Eloxx Pharmaceuticals, its developer, reported. The therapy was well-tolerated in single- and multiple-ascending dose studies in those clinical trials, and raised levels of the crucial CFTR protein in…
Claire’s Place Foundation is throwing an inaugural fundraising ball June 29 to celebrate the life and legacy of the foundation’s late founder, Claire Wineland, who had cystic fibrosis (CF). Called the Clairity Ball, the event will be held at the Fairmont Miramar Hotel & Bungalows in Santa Monica,…
Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in development for the treatment of cystic fibrosis (CF). This recognition of the therapeutic potential of PTI-428 in Europe follows previously granted orphan drug, breakthrough therapy, and fast…
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