Ontology highlight
ABSTRACT:
SUBMITTER: West M
PROVIDER: S-EPMC2678048 | biostudies-literature | 2009 May
REPOSITORIES: biostudies-literature
West Michael M Nicholls Kathy K Mehta Atul A Clarke Joe T R JT Steiner Robert R Beck Michael M Barshop Bruce A BA Rhead William W Mensah Robert R Ries Markus M Schiffmann Raphael R
Journal of the American Society of Nephrology : JASN 20090408 5
In male patients with Fabry disease, an X-linked disorder of glycosphingolipid metabolism caused by deficient activity of the lysosomal enzyme alpha-galactosidase A, kidney dysfunction becomes apparent by the third decade of life and invariably progresses to ESRD without treatment. Here, we summarize the effects of agalsidase alfa on kidney function from three prospective, randomized, placebo-controlled trials and their open-label extension studies involving 108 adult male patients. The mean bas ...[more]