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GR181413A

National Institutes of Health

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migalastat

Papers overview

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2014
2014
Phase 3 FACETS study of migalastat HCl for Fabry disease: post hoc GLA mutation-based identification of subjects likely to show a drug effect
Highly Cited
2011
Highly Cited
2011
A Pharmacogenetic Approach to Identify Mutant Forms of α-Galactosidase A that Respond to a Pharmacological Chaperone for Fabry Disease
Fabry disease is caused by mutations in the gene (GLA) that encodes α‐galactosidase A (α‐Gal A). The iminosugar AT1001 (GR181413A… 
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