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Long-term dose-dependent agalsidase effects on kidney histology in Fabry disease

Clinical Journal of the American Society of Nephrology Sep 23, 2017

Skrunes R, et al. - This study assessed the dose-dependent impacts of agalsidase therapy in serial kidney biopsies of patients with classic Fabry disease treated for up to 14 years. Podocyte globotriaosylceramide reduction was seen following prolonged treatment with different dosing regimens of agalsidase, correlating with cumulative dose. Limited clearing of arterial/arteriolar globotriaosylceramide raised concerns about long-term vascular impacts of current therapy. In men, a correlation was evident between residual plasma globotriaosylsphingosine and cumulative dose.
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