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Fabry disease (FD) is an X-linked lysosomal storage disorder (LSD) caused by mutations of the α-galactosidase A gene. The lysosomal enzyme α -galactosidase A (GLA) mediates the hydrolysis of the… Click to show full abstract
Fabry disease (FD) is an X-linked lysosomal storage disorder (LSD) caused by mutations of the α-galactosidase A gene. The lysosomal enzyme α -galactosidase A (GLA) mediates the hydrolysis of the terminal α -galactosyl moiety from globotriaosylceramide (Gb3). An enzymatic defect leads to the
Keywords:
replacement therapy;
therapy antidrug;
effects enzyme;
enzyme replacement;
fabry disease;
antidrug antibodies
Journal Title: Journal of the American Society of Nephrology : JASN
Year Published: 2018
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Journal Title: Journal of the American Society of Nephrology : JASN
Year Published: 2018
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!