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Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice

Approved therapies for Fabry disease (FD) include migalastat, an oral pharmacological chaperone, and agalsidase beta and agalsidase alfa, 2 forms of enzyme replacement therapy. Broad tissue distribution may be beneficial for clinical efficacy in FD, which has severe manifestations in multiple organs...

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Detalles Bibliográficos
Autores principales:	Wu, Yi Shuan, Khanna, Richie, Schmith, Virginia, Lun, Yi, Shen, Jin‐Song, Garcia, Anadina, Dungan, Leo, Perry, Anthony, Martin, Lukas, Tsai, Pai‐Chi, Hamler, Rick, Das, Anibh M., Schiffmann, Raphael, Johnson, Franklin K.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8453552/ https://www.ncbi.nlm.nih.gov/pubmed/33876577 http://dx.doi.org/10.1002/cpdd.941

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