Cystic Fibrosis 2023

https://www.thelancet.com/series/cystic-fibrosis-2023?dgcid=hubspot_email_conferencealerts_lanrescysticfibrosis23&utm_campaign=conferencealerts&utm_medium=email&_hsmi=273463263&_hsenc=p2ANqtz--ZRtX_yZmsvN73uuaygFcC-wJfQdDbPjD9sJYMI0Dyj0L_W_xIuatHURP_Zun26apaWWB8m0hSFyVnetrfjoO5_IkcDA&utm_content=273463263&utm_source=hs_email Cystic fibrosis is an autosomal recessive disorder caused by pathogenic variants of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The disease is thought to affect every organ system, with prominent pathology in the respiratory, gastrointestinal, endocrine, and reproductive systems. The development of modulators to restore CFTR function has led to substantial improvements in the health of many people with cystic fibrosis and transformed the landscape of research and clinical care. In the first of a Series of four papers, Jennifer Taylor-Cousar and colleagues describe the development of CFTR modulators, outlining key findings from clinical trials and highlighting the need for post-approval studies of longer-term safety and efficacy. Katherine Hisert and colleagues identify gaps in understanding and set out priorities for research to meet the needs of people with cystic fibrosis in the era of modulator therapy. Simon Graeber and Marcus Mall focus on the mechanisms of CFTR dysfunction, describing novel strategies for symptomatic therapy and promising approaches to the restoration of CFTR function. Finally, Nicole Mayer-Hamblett and colleagues present a new roadmap for the design and conduct of cystic fibrosis clinical trials that addresses the challenges of clinical research in the era of CFTR modulators.v