Original paper
Lyso-Gb3 associates with adverse long-term outcome in patients with Fabry disease
Abstract
Fabry disease (FD) is a rare X-linked lysosomal storage disease caused by mutations in the α-galactosidase A gene (GLA) leading to deficiency of α-galactosidase A and ultimately in progressive glycosphingolipid accumulation, especially globotriaosylceramide (Gb3) and its deacylated derivative globotriaosylsphingosine (Lyso-Gb3). The aim of the study was to assess plasma Lyso-Gb3 levels as a possible factor associated with adverse outcomes in...
Paper Details
Title
Lyso-Gb3 associates with adverse long-term outcome in patients with Fabry disease
Published Date
Jan 25, 2021
Journal
Volume
59
Issue
3
Pages
287 - 293
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Notes
History