Title : Mechanisms of Neutralizing Anti-drug Antibody Formation and Clinical Relevance on Therapeutic Efficacy of Enzyme Replacement Therapies in Fabry Disease.

Pub. Date : 2021 Nov

PMID : 34748189






1 Functional Relationships(s)
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1 The lysosomal accumulation of the substrates globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3) results in progressive renal failure, cardiomyopathy associated with cardiac arrhythmia, and recurrent strokes, significantly limiting life expectancy in affected patients. globotriaosylceramide alpha 1,4-galactosyltransferase (P blood group) Homo sapiens