CPIC guideline for ivacaftor therapy based on CFTR genotype of cystic fibrosis patients
Cystic fibrosis (CF) therapy has traditionally been based on the treatment of symptoms and infections, rather than targeting the underlying defect caused by variants in the CFTRgene. Ivacaftor is the first FDA-approved drug to specifically target a particular defect of the CFTR protein. It is indicated for use in patients