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Intestinal Current Measurements Detect Activation of Mutant CFTR in Cystic Fibrosis Patients with the G551D Mutation Treated with Ivacaftor

S Gräber, M Hug, O Sommerburg, S Hirtz, J Hentschel, A Heinzmann, C Dopfer, A Schulz, J Mainz, B Tümmler, MA Mall · Pneumologie · Ausgabe 07/2015

DOI 10.1055/s-0035-1556594

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Autor:innenS Gräber, M Hug, O Sommerburg, S Hirtz, J Hentschel, A Heinzmann, C Dopfer, A Schulz, J Mainz, B Tümmler, MA Mall
ZeitschriftPneumologie
Ausgabe07/2015
Jahrgang / SeitenNicht angegeben

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Abstract-Vorschau

Introduction: Recent studies suggest that sensitive outcome measures of in vivo CFTR activity may be important to accelerate clinical development of emerging CFTR modulator therapies for cystic fibrosis (CF). Intestinal current measurements (ICM) detect pharmacological activation of mutant CFTR ex vivo. However, whether ICM can detect rescue of mutant CFTR function in CF patients remains unknown. Objective: To deter…

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Zitierempfehlung

S Gräber, M Hug, O Sommerburg, S Hirtz, J Hentschel, A Heinzmann, C Dopfer, A Schulz, J Mainz, B Tümmler, MA Mall (2015) Intestinal Current Measurements Detect Activation of Mutant CFTR in Cystic Fibrosis Patients with the G551D Mutation Treated with Ivacaftor. Pneumologie (07). https://doi.org/10.1055/s-0035-1556594

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