Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
The University of Sydney · Westmead Hospital · +17 more institutions
Indexed incrossrefpubmed
Abstract
Background
mutation and a minimal-function mutation (Phe508del-minimal function genotype), the next-generation CFTR corrector elexacaftor, in combination with tezacaftor and ivacaftor, improved Phe508del CFTR function and clinical outcomes.
Methods
) at week 4.
Citation impact
2,086
total citations
- FWCI
- 157.57
- Percentile
- 100%
- References
- 38
Citations per year
Authors
21- PGPeter G. MiddletonCorresponding
The University of Sydney, Westmead Hospital, Westmead Institute for Medical Research
- MMMarcus Mall
German Center for Lung Research, Westmead Institute for Medical Research
- PDPavel Dřevı́nek
Charles University, Westmead Institute for Medical Research
- LCLarry C. Lands
McGill University Health Centre, Westmead Institute for Medical Research
- EFEdward F. McKone
University College Dublin, St. Vincent's University Hospital, Westmead Institute for Medical Research
Topics & keywords
Topics
Keywords
- Ivacaftor
- Cystic fibrosis
- Medicine
- Cystic fibrosis transmembrane conductance regulator
- Placebo
- Internal medicine
- Pathology
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