Clinical change 2 years from start of elexacaftor-tezacaftor-ivacaftor in severe cystic fibrosis.

Karen S McCoy,Jill Blind,Terri Johnson,Patti Olson,Laura Raterman,Shasha Bai,Mariah Eisner,Shahid I Sheikh,Stephan Druhan, Cody Young,Kimberly Pasley

Pediatric pulmonology（2023）

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摘要

This study focused on patients with CF eligible for modulator therapy and were initiated due to advanced lung disease. Initiation of modulator therapy was deemed safe and resulted in objective positive changes in nutrition, cough, FEV , subjective reports of clinical status, level of activity, and a reduction in burden of treatment.

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advanced lung disease,compassionate use,cystic fibrosis

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