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...
| Hlavní autoři: | , , , , , , , , , |
|---|---|
| Médium: | Conference item |
| Vydáno: |
Karger
2017
|