Lucerastat, an iminosugar for substrate reduction therapy in Fabry disease: preclinical evidence
Fabry disease (FD) is a lysosomal 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 neuropathic pa...
| Hlavní autoři: | , , , , , , , , , |
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| Médium: | Conference item |
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Elsevier
2017
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