Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial

P. Nichols, David; C. Paynter, Alex; L. Heltshe, Sonya; H. Donaldson, Scott; A. Frederick, Carla; D. Freedman, Steven; Gelfond, Daniel; R. Hoffman, Lucas; Kelly, Andrea; R. Narkewicz, Michael; E. Pittman, Jessica; Ratjen, Felix; Rosenfeld, Margaret; D. Sagel, Scott; Jane Schwarzenberg, Sarah; K. Singh, Pradeep; M. Solomon, George; S. Stalvey, Michael; P. Clancy, John; Kirby, Shannon; M. Van Dalfsen, Jill; H. Kloster, Margaret; M. Rowe, Steven

doi:10.1164/rccm.202108-1986oc

Public

Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial

Published in American Journal of Respiratory and Critical Care Medicine • Nov 16, 2021

Authors:

David P. Nichols

Alex C. Paynter

Sonya L. Heltshe

Abstract

Abstract Rationale The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved highly effective in controlled clinical trials for individuals with at least one F508del allele, which occurs in at least 85% of people with CF. Objectives PROMISE is a postapproval study to un...

Subject

Medicine

Ivacaftor

Cystic fibrosis

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