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migalastat hydrochloride

Known as: migalastat HCl 
National Institutes of Health

Papers overview

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2015
2015
Renal function may progressively decline in patients with Fabry disease. This study assessed pharmacokinetics, safety, and… 
2013
2013
Abstract Objective: Fabry disease is a rare X-linked disease caused by mutations to the GLA gene, resulting in a deficiency of… 
Highly Cited
2012
Highly Cited
2012
Fabry disease is an X-linked lysosomal storage disorder (LSD) caused by mutations in the gene (GLA) that encodes the lysosomal… 
Highly Cited
2012
Highly Cited
2012
BackgroundFabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A… 
2012
2012
Ferri L, Guido C, la Marca G, Malvagia S, Cavicchi C, Fiumara A, Barone R, Parini R, Antuzzi D, Feliciani C, Zampetti A, Manna R… 
Highly Cited
2011
Highly Cited
2011
Fabry disease is caused by mutations in the gene (GLA) that encodes α‐galactosidase A (α‐Gal A). The iminosugar AT1001 (GR181413A… 
Highly Cited
2009
Highly Cited
2009
SummaryFabry disease is an X-linked lysosomal storage disorder caused by mutations in the gene encoding α-galactosidase A (α-Gal…