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1.
Glucosylceramide and Glucosylsphingosine Quantitation by Liquid Chromatography-Tandem Mass Spectrometry to Enable In Vivo Preclinical Studies of Neuronopathic Gaucher Disease.
Anal Chem
; 89(16): 8288-8295, 2017 08 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-28686011
2.
Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice.
Mol Ther
; 20(4): 717-26, 2012 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-22215019
3.
Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice.
Clin Pharmacol Drug Dev
; 10(9): 1075-1088, 2021 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-33876577
4.
Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model.
PLoS One
; 16(6): e0252325, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-34106956
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