Should all children with cystic fibrosis who have responsive CFTR mutations be prescribed CFTR modulators?

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been a substantial advance in the treatment of patients with cystic fibrosis (CF). Modulators work through a process of chaperoning mutated CFTR to the epithelial membrane ( ‘corrector’) and improving the function of mutated CFTR at the epithelial surface (‘potentiator’) [1]. CFTR modulators are mutation specific so that not all mutations are responsive. However, the most common mutation (p.F508del, either homozygous or heterozygous) is responsive to a currently available triple combination therapy so that 90% of patients can now receive modulator therapy [2].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Review Source Type: research