Agalsidase benefits renal histology in young patients with Fabry disease.

  title={Agalsidase benefits renal histology in young patients with Fabry disease.},
  author={Camilla T\ondel and Leif Bostad and Kristin Kampevold Larsen and Asle Hirth and Bj\orn Egil Vikse and Gunnar Houge and Einar Svarstad},
  journal={Journal of the American Society of Nephrology : JASN},
  volume={24 1},
The effect of early-onset enzyme replacement therapy on renal morphologic features in Fabry disease is largely unknown. Here, we evaluated the effect of 5 years of treatment with agalsidase alfa or agalsidase beta in 12 consecutive patients age 7-33 years (median age, 16.5 years). We performed renal biopsies at baseline and after 5 years of enzyme replacement therapy; 7 patients had additional biopsies after 1 and 3 years. After a median of 65 months, biopsy findings from all patients showed… CONTINUE READING
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