Comment on “Translating Emerging Data for Fetal Treatment of Cystic Fibrosis”
Cystic fibrosis (CF) is a multisystem genetic disease caused by pathogenic variants in the cystic fibrosis transmembrane receptor ( CFTR ) gene, leading to abnormal function in the lungs, pancreas, intestines, and other organs. CFTR modulators (CFTRm), small molecule therapies for CF, have significantly improved...