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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Hlavní autoři:	Simona D’Amore, Sneha Patel, Juniebel Cooke, Uma Ramaswami
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	MDPI AG 2025-11-01
Edice:	Metabolites
Témata:	Gaucher disease lyso-Gb1 biomarker enzyme replacement therapy substrate reduction therapy
On-line přístup:	https://www.mdpi.com/2218-1989/15/11/731
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