Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data.
<h4>Objectives</h4>Cystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations. We used real-world eviden...
Main Authors: | Lisa J McGarry, Zahra Bhaiwala, Andrea Lopez, Conor Chandler, Christopher G Pelligra, Jaime L Rubin, Theodore G Liou |
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Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2023-01-01
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Series: | PLoS ONE |
Online Access: | https://doi.org/10.1371/journal.pone.0283479 |
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