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The lysosomal storage diseases (LSD) are a heterogeneous group of disorders, characterized by the progressive accumulation of various substrates in multiple cell types, as a consequence of defects in the degradation of by-products of cellular turnover. Several subtypes are associated with neurodegenerative features, which present as a major therapeutic challenge. Although the causal gene defects and...
Agalsidase alfa is a formulation of the human enzyme α-galactosidase A (AGAL), generated by activation of the encoding gene in a continuous human cell line. The regular infusion of agalsidase alfa into patients with Anderson-Fabry disease (AFD), in whom AGAL deficiency can lead to multi-organ system failure, has been demonstrated to be safe. By facilitating the clearance of the substrate globotriaosylceramide,...
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