The Present and Future of the Fabry Disease Market

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2025-09-13 17:55:13

Fabry disease represents a complex lysosomal storage pathology caused by GLA gene defects resulting in alpha-galactosidase A enzyme inadequacy. This metabolic dysfunction triggers toxic substrate accumulation, primarily globotriaosylceramide (Gb3), causing systematic organ deterioration across renal, cardiac, and neurological systems while profoundly impacting patient survival and...

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