Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis

Background Cystic fibrosis (CF) is a common, life‐shortening, genetic disorder in populations of Northern European descent caused by the mutation of a single gene that codes for the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This protein coordinates the tra...

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