The Cystic Fibrosis Foundation (CFF) is giving Eloxx Pharmaceuticals up to $1.61 million to support its planned Phase 2 clinical trial program assessing the safety, tolerability, and chemical properties of ELX-02, Eloxx’s lead investigational compound to treat cystic fibrosis (CF) caused by nonsense, or stop, mutations. The program…
CFTR channel activity is controlled by a molecular process known as phosphorylation. Now, a study suggests that the levels of phosphorylation affect how well Kalydeco works in people with certain mutations in the CFTR gene. The study “VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level” was…
Adults with cystic fibrosis (CF) are less active than they should be, although they have comparable activity levels to people without the genetic disorder, according to a recent review. The review also showed that there are not enough studies that objectively assess physical activity levels in CF. Therefore,…
The lung clearance index (LCI) is more sensitive to changes in lung function in patients with mild cystic fibrosis (CF) than conventional spirometry parameters, a study asserts. The findings were reported in the study, “Lung clearance index to detect the efficacy of Aztreonam lysine inhalation in…
A biosimilar of dornase alfa inhalation solution, with the same therapeutic activity as Pulmozyme, its reference therapy, was approved for use in Russia, Selexis and Generium Pharmaceutical announced. A…
These figures were taken from the Cystic Fibrosis Foundation’s (CFF) 2017 Patient Registry Annual Data Report. Share this infographic on your site! </p><br /> <p><strong>Please include attribution to https://cysticfibrosisnewstoday.com with…
Translate Bio is going to focus on the development of potential therapies to treat pulmonary diseases, including its lead candidate, MRT5005, targeting the underlying cause of cystic fibrosis (CF), the company announced. MRT5005 is designed to restore the levels of working cystic fibrosis transmembrane conductance regulator (CFTR) protein and reverse…
Prenatal genetic testing for cystic fibrosis (CF) significantly influences how women manage their pregnancies, with positive tests linked to high rates of pregnancy terminations, a systematic review study finds. The study, “Prenatal genetic testing for cystic fibrosis: a systematic review of clinical effectiveness and an ethics review,” was published…
Armata Pharmaceuticals is going to target multidrug-resistant Pseudomonas aeruginosa infections in cystic fibrosis (CF) patients with its new enhanced bacteriophage candidate AP-PA02, the company announced. AP-PA02 is being developed to replace…
A new agreement will give people in Scotland with cystic fibrosis access to the CF combination treatments Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) through the country’s health system, according to Vertex Pharmaceuticals, the company that sells both medicines. The announcement follows a prior rejection by the …
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