cystic fibrosis

NHS England and Vertex Pharmaceuticals have reached an agreement that will allow access to Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor), a triple combination therapy for cystic fibrosis (CF) to be used in a combination regimen with Kalydeco, as soon as the medication is approved by the European…

Anxiety related to fears of COVID-19 infection was higher among mothers of children with cystic fibrosis (CF) than those of healthy children, although teenage patients themselves were less anxious than age-matched peers without CF, a survey of families in Turkey reported. Mothers of young patients with chronic lung infections, and…

The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) as a treatment for cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and…

Nontuberculous mycobacteria (NTM), a type of infectious bacteria, is able to survive being dried for a full day, a study found, suggesting that drying is not a sufficient way to disinfect nebulizers used by people with cystic fibrosis (CF). The study, “Susceptibility of the Mycobacterium…

AR-501, an inhaled treatment candidate for chronic lung infections in people with cystic fibrosis (CF), has been deemed safe and well-tolerated in healthy volunteers, results from the first part of a Phase 1/2a clinical trial show. The trial’s safety monitoring committee (SMC) and a data safety monitoring board…

Renovion has raised $8.1 million in a financing round, supporting further development and planned clinical testing of ARINA-1, a nebulized treatment for chronic lung diseases like cystic fibrosis (CF) and non-CF bronchiectasis, and for lung transplants. “Patient and laboratory studies have…

Eloxx Pharmaceuticals has resumed enrollment in its Phase 2 clinical trial evaluating ELX-02 in cystic fibrosis (CF) patients in Israel and Europe, after it was temporarily halted due to the COVID-19 pandemic.  “We are pleased that enrollment in our Phase 2 clinical…

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