Abstract
The off-label use of elexacaftor/tezacaftor/ivacaftor (ETI) in 35 people with cystic fibrosis with the N1303K variant but not F508del was associated with increased lung function, which combined with ex vivo data, demonstrates that N1303K responds to ETI https://bit.ly/4asUXAx
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CITATION STYLE
Burgel, P. R., Sermet-Gaudelus, I., Girodon, E., Kanaan, R., Le Bihan, J., Remus, N., … Martin, C. (2024, January 1). Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint. European Respiratory Journal. European Respiratory Society. https://doi.org/10.1183/13993003.01959-2023
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