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Fabry Disease: The Current Treatment Landscape

Fabry disease (FD) is a rare X-linked lysosomal storage disease based on a deficiency of α-galactosidase A (AGAL) caused by mutations in the α-galactosidase A gene (GLA). The lysosomal accumulation of glycosphingolipids, especially globotriaosylceramide (Gb(3)) and globotriaosylsphingosine (lyso-Gb(...

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Bibliografiske detaljer
Udgivet i:Drugs
Main Authors: Lenders, Malte, Brand, Eva
Format: Artigo
Sprog:Inglês
Udgivet: Springer International Publishing 2021
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8102455/
https://ncbi.nlm.nih.gov/pubmed/33721270
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-021-01486-1
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