Ontology highlight
ABSTRACT:
SUBMITTER: Nichols DP
PROVIDER: S-EPMC8906485 | biostudies-literature | 2022 Mar
REPOSITORIES: biostudies-literature
Nichols David P DP Paynter Alex C AC Heltshe Sonya L SL Donaldson Scott H SH Frederick Carla A CA Freedman Steven D SD Gelfond Daniel D Hoffman Lucas R LR Kelly Andrea A Narkewicz Michael R MR Pittman Jessica E JE Ratjen Felix F Rosenfeld Margaret M Sagel Scott D SD Schwarzenberg Sarah Jane SJ Singh Pradeep K PK Solomon George M GM Stalvey Michael S MS Clancy John P JP Kirby Shannon S Van Dalfsen Jill M JM Kloster Margaret H MH Rowe Steven M SM
American journal of respiratory and critical care medicine 20220301 5
<b>Rationale:</b> 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. <b>Objectives:</b> PROMISE is a postapproval study to understand the broad effects of ETI through 30 months' clinical use in a more diverse U.S. patient population with planned analyses after 6 months. <b>Methods:</b> Prospective, observational stud ...[more]