Cystic Fibrosis Transmemb. Conduct. Reg. (CFTR) Correctors

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) correctors are a targeted therapeutic class of small-molecule CFTR modulators developed specifically for the management of cystic fibrosis These agents act as pharmacological chaperones that bind directly to misfolded CFTR proteins—most commonly associated with processing mutations such as the F508del allele—facilitating proper protein folding, stability, and intracellular trafficking to the apical cell membrane By increasing the density...

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) correctors are a targeted therapeutic class of small-molecule CFTR modulators developed specifically for the management of cystic fibrosis

These agents act as pharmacological chaperones that bind directly to misfolded CFTR proteins—most commonly associated with processing mutations such as the F508del allele—facilitating proper protein folding, stability, and intracellular trafficking to the apical cell membrane

By increasing the density of functional chloride and bicarbonate channels on epithelial surfaces, they help restore mucosal hydration and mucociliary clearance across pulmonary and gastrointestinal tissues, typically functioning in combination regimens with CFTR potentiators