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Roesel RA, Hommes FA, Samper L: Pyroglutamic aciduria (5-oxoprolinuria) without glutathione synthetase deficiency and with decreased pyroglutamate hydrolase activity

Supplementary Data 1 (download ZIP Atomic coordinates for starting, ending and selected time-points of MD simulations

doi: 10.1016/j.jcf.2019.08.020 15 BhatiaM.ZemansR

In a large number of in vitro studies, Lumacaftor showed good efficacy and tolerability, although the treatment by itself showed a significant decrease only in sweat chloride levels and no improvements in FEV 1 in a phase II trial with F508del-homozygous patients