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The effect of the glucosylceramide synthase inhibitor lucerastat on cardiac repolarization: results from a thorough QT study in healthy subjects

Abstract Background Fabry disease is a rare inherited glycosphingolipid storage disorder caused by deleterious mutations in the GLA gene coding for the lysosomal enzyme α-galactosidase A. The glucosylceramide synthase inhibitor lucerastat is an iminosugar with potential to provide oral substrate red...

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Main Authors: Markus S. Mueller, Patricia N. Sidharta, Christine Voors-Pette, Borje Darpo, Hongqi Xue, Jasper Dingemanse
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2020-10-01
Serier:Orphanet Journal of Rare Diseases
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Online adgang:http://link.springer.com/article/10.1186/s13023-020-01582-7
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