Enzyme Replacement Therapy in Fabry Disease.

Authors :: Schiffmann, Raphael
Kopp, Jeffrey B.
Austin III, Howard A.
Sabnis, Sharda
Moore, David F.
Weibel, Thais
Balow, James E.
Brady, Roscoe O.
Source :: JAMA: Journal of the American Medical Association. 6/6/2001, Vol. 285 Issue 21, p2743. 7p. 1 Diagram, 5 Charts, 1 Graph.
Publication Year :: 2001
Abstract: Reports on a study to evaluate the safety and efficacy of intravenous alpha-galactosidase A (alpha-gal A), a lysosomal enzyme, for Fabry disease. Causes of Fabry disease, a metabolic disorder which has no specific treatment; Methods; Results, including reduced severity of neuropathic pain in patients receiving alpha-gal A; Conclusions on the safety and efficacy of alpha-gal A.