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

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 th...

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Veröffentlicht in:Ann Am Thorac Soc
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Thoracic Society 2021
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Original Research
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8094270/
https://ncbi.nlm.nih.gov/pubmed/33095038
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1513/AnnalsATS.202006-659OC
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