Lucerastat, an iminosugar for substrate reduction therapy in Fabry Disease: Preclinical evidence

Lucerastat, an iminosugar for substrate reduction therapy in Fabry Disease: Preclinical evidence

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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...

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Bibliografiska uppgifter
Huvudupphovsmän: Welford, R, Mühleman, A, Priestman, D, Garzotti, M, Deymier, C, Ertel, E, Iglarz, M, Morand, O, Platt, F, Probst, M
Materialtyp: Conference item
Publicerad: Karger 2017

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