Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies

[摘要]：Areas covered: This paper discusses the results of a Phase II study investigating the safety and efficacy of VX-770 in patients with CF. Expert opinion: VX-770 is able to improve chloride and sodium transportation and has a good safety profile. Although such compounds have limited therapeutic targeting potential, preliminary results show great promise in the context of CF therapy.

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