Non-clinical evaluation of JR-051 as a biosimilar to agalsidase beta for the treatment of Fabry disease

Fabry disease (FD) is an X-linked lysosomal storage disease. It is caused by deficiency of the enzyme α-galactosidase A (α-Gal A), which leads to excessive deposition of neutral glycosphingolipids, especially globotriaosylceramide (GL-3), in cells throughout the body. Progressive accumulation of GL-...

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Veröffentlicht in:	Molecular genetics and metabolism 2018-09, Vol.125 (1-2), p.153-160
Hauptverfasser:	Morimoto, Hideto, Ito, Yae, Yoden, Eiji, Horie, Masato, Tanaka, Noboru, Komurasaki, Yoshikazu, Yamamoto, Ryuji, Mihara, Kazutoshi, Minami, Kohtaro, Hirato, Tohru
Format:	Artikel
Sprache:	eng
Schlagworte:	Agalsidase beta alpha-Galactosidase - administration & dosage alpha-Galactosidase - genetics Animals Biosimilar Biosimilar Pharmaceuticals - administration & dosage Enzyme Replacement Therapy Fabry disease Fabry Disease - drug therapy Fabry Disease - genetics Fabry Disease - pathology Fibroblasts Globotriaosylceramide Humans Isoenzymes - administration & dosage Isoenzymes - genetics Kidney - metabolism Kidney - pathology Liver - metabolism Liver - pathology Male Mannose-6-phosphate Mice Mice, Knockout Skin - metabolism Skin - pathology Spleen - metabolism Spleen - pathology Trihexosylceramides α-Galactosidase A
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