Ex Vivo and in Vivo Effects of Isofagomine on Acid β-Glucosidase Variants and Substrate Levels in Gaucher Disease

Autor Sun, Ying, Liou, Benjamin, Xu, You-Hai, Quinn, Brian, Zhang, Wujuan, Hamler, Rick, Setchell, Kenneth D. R., Grabowski, Gregory A.
Vydáno 2012

Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice

Autor Xu, Su, Lun, Yi, Brignol, Nastry, Hamler, Rick, Schilling, Adriane, Frascella, Michelle, Sullivan, Sean, Boyd, Robert E, Chang, Kate, Soska, Rebecca, Garcia, Anadina, Feng, Jessie, Yasukawa, Hidehito, Shardlow, Carole, Churchill, Alison, Ketkar, Amol, Robertson, Nicola, Miyamoto, Masahito, Mihara, Kazutoshi, Benjamin, Elfrida R, Lockhart, David J, Hirato, Tohru, Fowles, Susie, Valenzano, Kenneth J, Khanna, Richie
Vydáno v Mol Ther (2015)