Since 2012, drugs that target disease-causing variants in CFTR have become available to CF patients.9–11 For individuals with the F508del defect and those with some rare variants, combination therapy with two or more CFTR correctors (drugs that deliver variants to the cell membrane), together with a potentiator (drugs that boost channel activity), may prove at least partially effective.51–53 However, not all individuals living with CF are likely to benefit from combination therapy with CFTR modulators. The gene discussed is CFTR; the disease is cystic fibrosis.