Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

This review of 19 RCTs (n=2959) found there is insufficient evidence that corrector monotherapy has clinically important effects in pwCF with F508del/F508del. Further RCTs are required in children (under 12 years) and those with more severe respiratory function.

Source:

Cochrane Database of Systematic Reviews