The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts.

Mol Genet Metab Rep · 2019 · PMC6365982 · PMID 30775256

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near-significantno p-value reported

3.6.1.2 Agalsidase beta 1.0 mg/kg EOW With agalsidase beta, one single-arm CT publication reported a statistically significant improvement in pain scores (in 15 patients across all five dosing regimens followed for 2.5 months) [ 69 ], while another single-arm CT publication reported a near-significant reduction in pain intensity (in 13 patients followed for 5 months) [ 70 ].

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