Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis.

doi:10.1016/j.jcf.2022.10.002

Article

Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2023

Southern Kevin W, Castellani Carlo, Lammertyn Elise, Smyth Alan, VanDevanter Donald, van Koningsbruggen-Rietschel Silke, Barben Jürg, Bevan Amanda, Brokaar Edwin, Collins Sarah, Connett Gary J, Daniels Thomas W V, Davies Jane, Declercq Dimitri, Gartner Silvia, Gramegna Andrea, Hamilton Naomi, Hauser Jenny, Kashirskaya Nataliya, Kessler Laurence, Lowdon Jacqueline, Makukh Halyna, Martin Clémence, Morrison Lisa, Nazareth Dilip, Noordhoek Jacquelien, O'Neill Ciaran, Owen Elizabeth, Oxley Helen, Raraigh Karen S, Raynal Caroline, Robinson Karen, Roehmel Jobst, Schwarz Carsten, Sermet Isabelle, Shteinberg Michal, Sinha Ian, Takawira Constance, van Mourik Peter, Verkleij Marieke, Waller Michael D, Duff Alistair

Abstract excerpt

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF. The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators...

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Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis.

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