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Migalastat: A Review in Fabry Disease

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 of α-galactosidase A enzyme from t...

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Detalles Bibliográficos
Publicado en:Drugs
Main Authors: McCafferty, Emma H., Scott, Lesley J.
Formato: Artigo
Idioma:Inglês
Publicado: Springer International Publishing 2019
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6647464/
https://ncbi.nlm.nih.gov/pubmed/30875019
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-019-01090-4
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