Question

Based on its mechanism of action, what would you predict to be the effect of treating a CFG551D patient with just Lumacaftor (VX-809)?

A. Lumacaftor would have a positive therapeutic outcome by reducing ERAD-ication of CFTRG551D resulting in higher levels of CFTRG551D on the apical membrane.

B. Lumacaftor would not have a positive therapeutic outcome because CFTRG551D would still be ERADicated resulting in too little CFTRG551D on the apical membrane.

C. Lumacaftor would not have a positive therapeutic outcome because CFTRG551D is not ERAD-icated, but cannot effectively transport Cl- across the apical membrane because it is defective in gating.

D. Lumacaftor would have at least some positive therapeutic outcome by increasing CFTRG551D gating so it could more efficiently transport Cl- across the apical membrane, but since it would not reduce ERAD-ication, there would not be a net increase in the level of CFTRG551D on the apical membrane.

Answer 1

a, Lumacrafter would have a positive therapeutic outcome by reducing ERAD-ication of CFTRG551D resulting in a higher level of CFTRG551D on the apical membrane.
Lumacrafter -ivacaftor for treating homozygous patients cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. it improves pulmonary function, reduced pulmonary exacerbation and has a safe positive risk-benefit ratio. the effect of VX-809 restore CFTR function and achieve ivacaftor function. Lumacrafter ivacaftor provide good evidence on clinically outcome.