The advent of high efficacy modulator therapies (HEMT) of CFTR has transformed cystic fibrosis care and also changed the biology of the disease. However, lung function can continue to decline following HEMT. The molecular basis for disease progression in people with CF on HEMT is not understood. Clinical and molecular biomarkers following therapy remain important to predict disease progression and/or point to disease-associated pathways that may be sites of intervention. Furthermore, biomarkers can aid in identifying patients that would derive maximum benefit from HEMT and those that may be good candidates for therapy simplification.
Originally recorded November 5, 2022.
