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Volume 2 Issue 1

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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation

Ramsey BW, Davies J, McElvaney NG et al.
Seattle Children’s Hospital, Seattle WA, USA.
N Engl J Med 2011;365:1663–72.
Editor’s note: Cystic fibrosis transmembrane conductance regulator (CFTR) mutations affect either the quantity of CFTR protein reaching the cell surface or the function of that protein once it arrives. G551D CFTR mutations affect the function of CFTR at the cell surface and, as such, may respond to potentiator agents that are designed to increase the duration that the CFTR channel remains open once activated. One such agent that has shown benefit in Phase I and II studies is ivacaftor (or VX-770).
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