No abstract available
Keywords:
Ataluren; cystic fibrosis; gene therapy; ivacaftor; lumacaftor ▪.
MeSH terms
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Aminophenols / therapeutic use
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Aminopyridines / therapeutic use
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Benzodioxoles / therapeutic use
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Cystic Fibrosis / genetics*
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Cystic Fibrosis / therapy*
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Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
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Genetic Therapy
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Humans
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Molecular Targeted Therapy
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Mutation*
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Oxadiazoles / therapeutic use
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Quinolones / therapeutic use
Substances
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Aminophenols
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Aminopyridines
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Benzodioxoles
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CFTR protein, human
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Oxadiazoles
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Quinolones
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Cystic Fibrosis Transmembrane Conductance Regulator
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ivacaftor
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lumacaftor
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ataluren