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Clinical relevance of globotriaosylceramide accumulation in Fabry disease and the effect of agalsidase beta in affected tissues [PDF]
Molecular Genetics and Metabolism, 2022 Fabry disease (FD) is a rare lysosomal storage disorder, characterized by a reduction in α-galactosidase A enzyme activity and the progressive accumulation of globotriaosylceramide (GL3) and its metabolites in the cells of various organs. Agalsidase beta,
Nicole Lyn +2 more
exaly +2 more sources
Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: A pilot study [PDF]
Kidney International, 2004 Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: A pilot study.BackgroundWe sought to assess the safety and efficacy of enzyme replacement therapy (ERT) with recombinant human-α-galactosidase A (rh-α-Gal A)
Vincenzo Panichi +2 more
exaly +2 more sources
Switch to agalsidase alfa after shortage of agalsidase beta in Fabry disease: a systematic review and meta-analysis of the literature [PDF]
Genetics in Medicine, 2017 Background:In 2009, the agalsidase beta shortage resulted in switching to agalsidase alfa treatment for many Fabry disease patients, offering the unique opportunity to compare the effects of the two drugs.
Massimo Imbriaco +2 more
exaly +2 more sources
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