Ontology highlight
ABSTRACT:
SUBMITTER: Fervenza FC
PROVIDER: S-EPMC2727881 | biostudies-other | 2008 Dec
REPOSITORIES: biostudies-other
Biologics : targets & therapy 20081201 4
Kidney involvement with progressive loss of kidney function (Fabry nephropathy) is an important complication of Fabry disease, an X-linked lysosomal storage disorder arising from deficiency of alpha-galactosidase activity. Clinical trials have shown that enzyme replacement therapy (ERT) with recombinant human alpha-galactosidase clears globotriaosylceramide from kidney cells, and can stabilize kidney function in patients with mild to moderate Fabry nephropathy. Recent trials show that patients w ...[more]