Your institution may have access to this item. Find your institution then sign in to continue.
Title
Enzyme replacement therapy in Fabry disease: a randomized controlled trial.
Authors
Schiffmann, R; Kopp, J B; Austin, H A, 3rd; Sabnis, S; Moore, D F; Weibel, T; Balow, J E; Brady, R O
Abstract
Fabry disease is a metabolic disorder without a specific treatment, caused by a deficiency of the lysosomal enzyme alpha-galactosidase A (alpha-gal A). Most patients experience debilitating neuropathic pain and premature mortality because of renal failure, cardiovascular disease, or cerebrovascular disease.