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Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein are called CFTR modulators. The two main types of modulators are potentiators and correctors.

Theratyping matches therapies, or medications, to specific types of mutations. The primary goal of theratyping is to identify which mutations respond to certain CFTR modulators, thereby helping people with rare CFTR mutations gain access to already approved modulators quickly and safely.