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Article

Ivacaftor in People with Cystic Fibrosis and a 3849+10kb C→T or D1152H Residual Function Mutation.

Annals of the American Thoracic Society - 1 Mar 2021

Kerem Eitan, Cohen-Cymberknoh Malena, Tsabari Reuven, Wilschanski Michael, Reiter Joel, Shoseyov David, Gileles-Hillel Alex, Pugatsch Thea, Davies Jane C, Short Christopher, Saunders Clare, DeSouza Cynthia, Sullivan James C, Doyle Jamie R, Chandarana Keval, Kinnman Nils

Abstract excerpt

Rationale: Ivacaftor's clinical effects in the residual function mutations 3849 + 10kb C→T and D1152H warrant further characterization.Objectives: To evaluate ivacaftor's effect in people with cystic fibrosis aged ≥6 years with 3849 + 10kb C→T or D1152H residual function mutations and to explore the correlation between ivacaftor-induced organoid-based cystic fibrosis transmembrane conductance regulator function...

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Identifiers

PMID: 33095038
DOI: 10.1513/AnnalsATS.202006-659OC
PMCID: PMC8094270

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