• Jennifer L Taylor-Cousar, Paul D Robinson, Michal Shteinberg, and Damian G Downey.
• Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Health, Denver, CO, USA; Division of Pediatric Pulmonary Medicine, National Jewish Health, Denver, CO, USA; Division of Pulmonary Sciences and Critical Care Medicine, Anschutz Medical Campus, University of Colorado, Aurora, CO, USA. Electronic address: taylorcousarj@NJHealth.org.
• Lancet. 2023 Sep 30; 402 (10408): 117111841171-1184.

AbstractFollowing discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that result, a new era of cystic fibrosis management has emerged-one in which scientific principles translated from the bench to the bedside have enabled us to potentially treat the basic defect in the majority of children and adults with cystic fibrosis, with a resultant burgeoning adult cystic fibrosis population. However, the long-term effects of these therapies on the multiple manifestations of cystic fibrosis are still under investigation. Understanding the effects of modulators in populations excluded from clinical trials is also crucial. Furthermore, establishing appropriate disease measures to assess efficacy in the youngest potential trial participants and in those whose post-modulator lung function is in the typical range for people without chronic lung disease is essential for continued drug development. Finally, recognising that a health outcome gap has been created for some people and widened for others who are not eligible for, cannot tolerate, or do not have access to modulators is important.Copyright © 2023 Elsevier Ltd. All rights reserved.

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