Fabry Disease Treatments 2025: What's Ahead in the Pipeline

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2025-06-17 10:53:54

Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, leading to a deficiency of the enzyme alpha-galactosidase A (α-Gal A). This enzyme is responsible for breaking down globotriaosylceramide (GL-3 or Gb3), a complex lipid. Enzyme deficiency results in systemic accumulation of Gb3 and related glycosphingolipids, particularly in vascular...

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