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Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR A Clinical Trial

Sagel, SD; Khan, U; Heltshe, SL; Clancy, JP; Borowitz, D; Gelfond, D; Donaldson, SH; Moran, A; Ratjen, F; VanDalfsen, JM; Rowe, SM

Sagel, SD (corresponding author), Childrens Hosp Colorado, 13123 East 16th Ave,B-395, Aurora, CO 80045 USA.

ANNALS OF THE AMERICAN THORACIC SOCIETY, 2021; 18 (1): 75

Abstract

Rationale: The combination of lumacaftor (LUM) and ivacaftor (IVA) is an approved CFTR (cystic fibrosis [CF] transmembrane conductance regulator) modu......

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