Small molecular therapies for cystic fibrosis (MIM: 602421) are well studied and include ivacaftor, which increases the time fraction that the cystic fibrosis transmembrane conductance regulator (CFTR) channel remains open, and lumacaftor, which increases the amount of CFTR that reaches the cell surface (Wainwright et al., 2015; Habib et al., 2019). This evidence concerns the gene CFTR and cystic fibrosis.