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migalastat hydrochloride

Known as: migalastat HCl 
National Institutes of Health

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AT-1001
migalastat

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
2018
2018
Résultats cardiaques au long cours du traitement par migalastat chez des patients atteints de la maladie de Fabry : résultats des essais cliniques de Phase 3
Review
2018
Review
2018
Treatment in Fabry disease.
2018
2018
Treatment in Fabry disease
  • M. L. Rodríguez
  • Revista Clínica Española (English Edition)
  • 2018
  • Corpus ID: 135469550
2015
2015
An open‐label study to determine the pharmacokinetics and safety of migalastat HCl in subjects with impaired renal function and healthy subjects with normal renal function
Renal function may progressively decline in patients with Fabry disease. This study assessed pharmacokinetics, safety, and… 
Highly Cited
2013
Highly Cited
2013
A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects.
Highly Cited
2012
Highly Cited
2012
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
BackgroundFabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A… 
2012
2012
Fabry disease: polymorphic haplotypes and a novel missense mutation in the GLA gene
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
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… 
2011
2011
A pharmacogenetic analysis of Fabry patient responses to pharmacological chaperone treatment with AT1001 (migalastat hydrochloride)
Highly Cited
2009
Highly Cited
2009
The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines
SummaryFabry disease is an X-linked lysosomal storage disorder caused by mutations in the gene encoding α-galactosidase A (α-Gal… 
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