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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological chaperone for the treatment of α-galactosidase A (α-Gal A) deficiency, which leads to Fabry disease, an X-linked, lysosomal storage disorder. The currently approved, biologics-based therapy for Fabry disease is...

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Detalles Bibliográficos
Autores principales:	Warnock, David G., Bichet, Daniel G., Holida, Myrl, Goker-Alpan, Ozlem, Nicholls, Kathy, Thomas, Mark, Eyskens, Francois, Shankar, Suma, Adera, Mathews, Sitaraman, Sheela, Khanna, Richie, Flanagan, John J., Wustman, Brandon A., Barth, Jay, Barlow, Carrolee, Valenzano, Kenneth J., Lockhart, David J., Boudes, Pol, Johnson, Franklin K.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2015
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4529213/ https://www.ncbi.nlm.nih.gov/pubmed/26252393 http://dx.doi.org/10.1371/journal.pone.0134341

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