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In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

27 Mar 2019

Abstract excerpt

animals showed the greatest VX-770-mediated protection from these pathologies. Sustained postnatal VX-770 administration led to improved pancreatic exocrine function, glucose tolerance, growth and survival, and to reduced mucus accumulation and bacterial infections in the lung. VX-770 withdrawal at any age reestablished disease, with the most rapid onset of morbidity occurring when withdrawal was initiated during...

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PMID: 30918114
DOI: 10.1126/scitranslmed.aau7531

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