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Figure 1 | Orphanet Journal of Rare Diseases

Figure 1

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 1

PBMC α-Gal A activity (as a percentage of normal) by week of migalastat HCl treatment. The red lines are for patients with mutations amenable to migalastat HCl as measured in the HEK-293 cell-based assay. The blue lines are for patients with non-responsive mutations. Values below 0.2 are below the limit of quantification (BLQ).

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172