Lucerastat, an iminosugar for substrate reduction therapy in Fabry Disease: Preclinical evidence
Fabry disease (FD) is a lysosomal storage disorder caused by mutations in the GLA gene coding for α-galactosidase A (α-GalA). These mutations lead to the accumulation of α-GalA substrates, including globotriaosylceramide (Gb3). As a consequence of lipid storage, Fabry patients can suffer from neurop...
المؤلفون الرئيسيون: | Welford, R, Mühleman, A, Priestman, D, Garzotti, M, Deymier, C, Ertel, E, Iglarz, M, Morand, O, Platt, F, Probst, M |
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التنسيق: | Conference item |
منشور في: |
Karger
2017
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مواد مشابهة
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Lucerastat, an iminosugar for substrate reduction therapy in Fabry disease: preclinical evidence
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The effect of the glucosylceramide synthase inhibitor lucerastat on cardiac repolarization: results from a thorough QT study in healthy subjects
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