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The era of mutation-specific therapies truly started with the FDA and EMA approval of the CFTR potentiator Ivacaftor in patients bearing at least one copy of G551D or some other class III mutation

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[DOI] [PMC free article] [PubMed] [Google Scholar] [97].Wang X, Vatamaniuk MZ, Roneker CA, Pepper MP, Hu LG, Simmons RA, Lei XG, Knockouts of SOD1 and GPX1 exert different impacts on murine islet function and pancreatic integrity

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