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Figure 2 | Orphanet Journal of Rare Diseases

Figure 2

From: Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Figure 2

Total urine GL-3 (in pmol/nmol phosphotidlycholine) by week of migalastat HCl treatment. The red lines are for patients with an AT1001-amenable mutation in the HEK-293 cell-based assay, and the blue lines are for those with non-amenable mutations (Mutation p.F295C in patient 3–0301) was responsive only when high concentrations of migalastat HCl where used). Y axis: logarithmic scale.

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172