In terms of beneficence, there is compelling real-world evidence of substantial benefits of CFTRm therapy in pwCF and responsive CFTR mutations, with reported improvements in lung function and nutrition, reduced pulmonary exacerbation rates as well as the potential to alter the trajectory of CF disease, and low mortality rates in the long term (Benden and Schwarz, 2021; Balfour-Lynn and King, 2022; Gifford et al., 2022; Keogh et al., 2022; Regard et al., 2022; Sawicki et al., 2015; Heijerman et al., 2019; Volkova et al., 2020; Carnovale et al., 2022). This evidence concerns the gene CFTR and cystic fibrosis.