Treatment effects of CFTR modulators on people with Cystic Fibrosis carrying the Q359K/T360K variant

The identification of the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene and CF-causing variants has led to the development of therapies to correct the basic cellular defect. CFTR modulators (CFTRm) are such therapies that either correct protein misfolding (correctors) or restore cell surface channel activity (potentiators) [1, 2]. Footnotes This manuscript has recently been accepted for publication in the ERJ Open Research . It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJOR online. Please open or download the PDF to view this article. Competing interests: Author MS declares grants from GSK, Trudell, Tel Aviv league for lung disease during the conduct of the study; consulting fees from Astra Zeneca, Boehringer Ingelheim, Dexcel, Kamada, Synchrony medical, Trumed, Vertex, Zambon; Payment for lectures fees from Astra Zeneca, Boehringer Ingelheim, GSK, Kamada, Sanofi, Insmed; support for attending meetings fees from Astra Zeneca, Boehringer Ingelheim, Kamada, Rafa, GSK; participation on advisory board fees from Bonus Biotherapeutics, Boehringer Ingelheim, Astra Zeneca; Leadership in board fees from AJRCCM; Receipt of equipment from Trudell outside the submitted work. Competing interests: Author DP declares grants, consulting fees and payment of lectures from Vertex, outside the submitted work Competing interests: Author KY declares grants and support for attending meetings fees from Sanofi outside the submitted work. Competing interests:Author RC declares payment of lectures from GSK and support for attending meetings fees from Boehringer Ingelheim, Astra Zeneca, Kamada outside the submitted work. Competing interests: Authors MH, MRK, ES, FV, ASR, GL declare that they have no conflict of interest.