RECOMB HUMAN ALPHA GALACTOSIDASE IN FABRY DISEASE

$0M01FY2000RRNIH

Beth Israel Deaconess Medical Center, Boston MA

Investigators

Linked publications & trials

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Abstract

Primary objectives of this study are: -To demonstrate the long term safety of 4-h(alpha)GAL in patients with Fabry disease as determined by adverse experiences/events (AEs), vital signs, ECGs, echocardiograms, and clinical laboratory parameters. -To demonstrate the continued efficacy of r-h(alpha)GAL in patients with Fabry disease in reducing GL-3 from the capillary endothelium of kidney, heart and skin tissue as measured by histological observation and biochemical measurement of GL-3 tissue samples from all three organs.

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