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Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria.
BACKGROUND Information concerning renal pathological characteristics in Fabry disease in childhood is limited. Our objective is to define renal morphological abnormalities in children and adolescentsExpand
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Safety and complications of percutaneous kidney biopsies in 715 children and 8573 adults in Norway 1988-2010.
BACKGROUND AND OBJECTIVES Skepticism about performing renal biopsies is often because of uncertainty regarding risk of complications. The aim of this study was to evaluate safety and relevantExpand
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Agalsidase benefits renal histology in young patients with Fabry disease.
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 orExpand
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Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
IntroductionFabry disease (FD) is a lysosomal storage disorder resulting in progressive nervous system, kidney and heart disease. Enzyme replacement therapy (ERT) may halt or attenuate diseaseExpand
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Reaccumulation of globotriaosylceramide in podocytes after agalsidase dose reduction in young Fabry patients
Background Agalsidase-α 0.2 mg/kg every other week (eow) and agalsidase-β 1.0 mg/kg/eow are licensed in Europe as equipotent treatment of the α-galactosidase deficiency in Fabry disease. This caseExpand
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Longevity of B-Cell and T-Cell Responses After Live Attenuated Influenza Vaccination in Children
Background. The live attenuated influenza vaccine (LAIV) is the preferred vaccine for children, but the mechanisms behind protective immune responses are unclear, and the duration of immunity remainsExpand
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Characterization of Early Disease Status in Treatment-Naive Male Paediatric Patients with Fabry Disease Enrolled in a Randomized Clinical Trial
Trial Design This analysis characterizes the degree of early organ involvement in a cohort of oligo-symptomatic untreated young patients with Fabry disease enrolled in an ongoing randomized,Expand
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The MDRD equation may mask decline of glomerular filtration rate in Fabry patients with normal or nearly normal kidney function.
AIMS The renal prognosis in Fabry disease is better when enzyme replacement therapy (ERT) is initiated before glomerular filtration rate (GFR) deteriorates. Current studies evaluating kidney functionExpand
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One Year of Enzyme Replacement Therapy Reduces Globotriaosylceramide Inclusions in Podocytes in Male Adult Patients with Fabry Disease
Fabry nephropathy is associated with progressive accumulation of globotriaosylceramide (GL3) in podocytes. Reducing this GL3 burden may reduce podocyte injury. Sensitive methods to quantify podocyteExpand
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