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-  2018 

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

DOI: 10.1126/scitranslmed.aau7531

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Abstract:

In utero and early postnatal functional rescue of cystic fibrosis transmembrane conductance regulator improves survival in cystic fibrosis ferrets

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