Combination lumacaftor and ivacaftor therapy for cystic fibrosis

References

Papers of special note have been highlighted as either of interest (•) or of considerable interest (••) to readers

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•• This is the pivotal clinical trial of ivacaftor vs. placebo in CF mediated by the G551D CFTR mutation.

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• This paper describes the physiologic effects of lumacaftor in vivo.

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• This is the pivotal phase 3 trial of lumacaftor-ivacaftor therapy for F508del homozygous cystic fibrosis.

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