Agalsidase benefits renal histology in young patients with Fabry disease
C Tøndel, L Bostad, KK Larsen, A Hirth… - Journal of the …, 2013 - journals.lww.com
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 total clearance of glomerular …
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 total clearance of glomerular …