Migalastat: A Review in Fabry Disease
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DOI: 10.1007/s40265-019-01090-4
Abstract: Fabry disease is a rare lysosomal disorder characterized by deficient or absent α-galactosidase A activity resulting from mutations in the GLA gene. Migalastat (Galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of amenable mutant forms… read more here.
Keywords: migalastat; amenable gla; fabry disease; disease migalastat ... See more keywords