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Article

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

Annals of the American Thoracic Society - 1 Jan 2021

Sagel Scott D, Khan Umer, Heltshe Sonya L, Clancy John P, Borowitz Drucy, Gelfond Daniel, Donaldson Scott H, Moran Antoinette, Ratjen Felix, VanDalfsen Jill M, Rowe Steven M

Abstract excerpt

Rationale: The combination of lumacaftor (LUM) and ivacaftor (IVA) is an approved CFTR (cystic fibrosis [CF] transmembrane conductance regulator) modulator treatment for homozygous F508del patients with CF.Objectives: To evaluate the effectiveness of LUM/IVA in children (6 yr or more) and adults (more than 18 yr) in a postapproval setting.Methods: This longitudinal cohort study, performed at 38 centers in the...

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Identifiers

PMID: 32644818
DOI: 10.1513/AnnalsATS.202002-144OC
PMCID: PMC7780982

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