Cargando…
Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb(3)) in Fabry Transgenic Mice and in the Plasma of Fabry Patients
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A (α-Gal A), and involves pathological accumulation of globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb(3)). Migalastat hydrochloride (GR181413A) is a pharmacological chape...
Autores principales: | Young-Gqamana, Brandy, Brignol, Nastry, Chang, Hui-Hwa, Khanna, Richie, Soska, Rebecca, Fuller, Maria, Sitaraman, Sheela A., Germain, Dominique P., Giugliani, Roberto, Hughes, Derralynn A., Mehta, Atul, Nicholls, Kathy, Boudes, Pol, Lockhart, David J., Valenzano, Kenneth J., Benjamin, Elfrida R. |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2013
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3589404/ https://www.ncbi.nlm.nih.gov/pubmed/23472096 http://dx.doi.org/10.1371/journal.pone.0057631 |
Ejemplares similares
-
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
por: Benjamin, Elfrida R., et al.
Publicado: (2017) -
Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease
por: Bichet, Daniel G., et al.
Publicado: (2020) -
Correction: Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease
por: Bichet, Daniel G., et al.
Publicado: (2020) -
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
por: Warnock, David G., et al.
Publicado: (2015) -
The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease
por: Schiffmann, Raphael, et al.
Publicado: (2019)