Abstract

introduction Fabry disease is a rare metabolic storage disorder that results in progressive effects on multiple organs; clinical manifestations include pain from nerve damage, gastrointestinal symptoms, angiokeratoma, corneal changes, and hearing loss.1,2 Over time, more serious manifestations develop, including renal dysfunc-tion, cardiac disorders such as cardiomyopathy and arrhyth-mias, and cerebrovascular events such as strokes, which all contribute to a reduced life expectancy.1,2 The treatment options for patients with Fabry disease include long-term enzyme-replacement therapy (ERT) in addition to supportive manage-ment.1 ERT using recombinant human α-galactosidase A is intended to reduce disease severity and delay the progression of the disorder.3,4 Two preparations for treatment are available, namely, agalsidase-α (Replagal) and agalsidase-β (Fabrazyme)

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