Gaucher Disease—Correlation of Lyso-Gb1 with Haematology and Biochemical Parameters
<b>Background/Objectives</b>: Gaucher disease (GD) is a lysosomal disorder caused by a deficiency of β-glucosidase. Disease-modifying therapies (DMTs) include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Glucosylsphingosine (lyso-Gb1) is a biomarker with high sensitivity a...
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| Autori principali: | , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
MDPI AG
2025-11-01
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| Serie: | Metabolites |
| Soggetti: | |
| Accesso online: | https://www.mdpi.com/2218-1989/15/11/731 |
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