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Differential effects of (S)- and (R)-vanzacaftor on BKCa potentiation and CFTR correction () - Sep 4, 2024 - Abstract #NACFC2024NACFC_1016; Incubation in (S)-VX-121 converts a population of band B F508del CFTR to band C, consistent with F508del CFTR correction, but (R)-VX-121 fails to correct F508del CFTR. (S)- and (R)-VX-121 both stimulate K+ secretion across HBECs and potentiate BKCa channels during whole-cell patch-clamp recording.
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Trial completion, Combination therapy: A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis (clinicaltrials.gov) - Jan 9, 2020 P2, N=87, Completed, This, alongwith more readily accessible material of a wide genotype range and greaterexpansion capability, makes the hPEM model an attractive platform forassessing CFTR functional rescue in response to novel therapeutic agents. Active, not recruiting --> Completed
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