Fabry disease

Effects of agalsidase-β shortage in Fabry disease

    A shortage in agalsidase-β between 2009 and 2012 resulted in patients with Fabry disease either receiving reduced doses or switching to agalsidase-α. Lenders et al. found that patients who reduced doses or switched medications exhibited a decline in renal function compared to those who received the standard agalsidase-β treatment regimen. All patients, however, showed a stable clinical disease course.


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      Lenders, M. et al. Patients with Fabry disease after enzyme replacement therapy dose reduction and switch-2-year follow-up. J. Am. Soc. Nephrol. 10.1681/ASN.2015030337

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    Effects of agalsidase-β shortage in Fabry disease. Nat Rev Nephrol 11, 566 (2015). https://doi.org/10.1038/nrneph.2015.139

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