Summary

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• Fabrazyme (agalsidase beta) is indicated for the treatment of adult and pediatric patients aged 2 years and older with confirmed Fabry disease.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• ERT with agalsidase beta significantly decreases globotriaosylceramide levels in various biological compartments, including plasma, urine, and specific kidney, heart, and skin cell types.
• ERT with agalsidase beta slows the decline in estimated glomerular filtration rate (eGFR), suggesting a potential protective effect on renal function, and improves cardiac metrics by reducing or stabilizing left ventricular mass and cardiac wall thickness.
• In adult male patients, particularly those who begin treatment early, ERT with agalsidase beta shows significant clinical benefits, including improvements in nervous system outcomes, pain management, gastrointestinal symptoms, and overall quality of life.
• There is no safety information available in the reviewed studies.
• There is no population type or subgroup information available in the reviewed studies.