With the aim to search for appropriate pharmacological approaches to correct the dysfunction of mutant CFTR, CF research community has been focusing its attention on small molecules able (i) to promote the delivery of the defective channel to the plasma membrane (namely correctors) by direct binding to CFTR or to other proteins involved in the protein trafficking machinery, and/or (ii) to improve ions permeation through CFTR (namely potentiators) by binding to NBDs or MSDs. The gene discussed is CFTR; the disease is cystic fibrosis.