The role of glucosylsphingosine as an early indicator of disease progression in early symptomatic type 1 Gaucher disease
Gaucher disease (GD), a lysosomal storage disorder caused by β-glucocerebrosidase deficiency, results in the accumulation of glucosylceramide and glucosylsphingosine. Glucosylsphingosine has emerged as a sensitive and specific biomarker for GD and treatment response. However, limited information exi...
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| Principais autores: | , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Elsevier
2021-06-01
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| Serier: | Molecular Genetics and Metabolism Reports |
| Fag: | |
| Online adgang: | http://www.sciencedirect.com/science/article/pii/S2214426921000239 |
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