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Improved efficacy of a next-generation ERT in murine Pompe disease.
Xu S, Lun Y, Frascella M, Garcia A, Soska R, Nair A, Ponery AS, Schilling A, Feng J, Tuske S, Valle MCD, Martina JA, Ralston E, Gotschall R, Valenzano KJ, Puertollano R, Do HV, Raben N, Khanna R. Xu S, et al. Among authors: garcia a. JCI Insight. 2019 Mar 7;4(5):e125358. doi: 10.1172/jci.insight.125358. eCollection 2019 Mar 7. JCI Insight. 2019. PMID: 30843882 Free PMC article.
The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease.
Khanna R, Powe AC Jr, Lun Y, Soska R, Feng J, Dhulipala R, Frascella M, Garcia A, Pellegrino LJ, Xu S, Brignol N, Toth MJ, Do HV, Lockhart DJ, Wustman BA, Valenzano KJ. Khanna R, et al. Among authors: garcia a. PLoS One. 2014 Jul 18;9(7):e102092. doi: 10.1371/journal.pone.0102092. eCollection 2014. PLoS One. 2014. PMID: 25036864 Free PMC article.
Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice.
Xu S, Lun Y, Brignol N, Hamler R, Schilling A, Frascella M, Sullivan S, Boyd RE, Chang K, Soska R, Garcia A, Feng J, Yasukawa H, Shardlow C, Churchill A, Ketkar A, Robertson N, Miyamoto M, Mihara K, Benjamin ER, Lockhart DJ, Hirato T, Fowles S, Valenzano KJ, Khanna R. Xu S, et al. Among authors: garcia a. Mol Ther. 2015 Jul;23(7):1169-1181. doi: 10.1038/mt.2015.87. Epub 2015 Apr 27. Mol Ther. 2015. PMID: 25915924 Free PMC article.
Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice.
Wu YS, Khanna R, Schmith V, Lun Y, Shen JS, Garcia A, Dungan L, Perry A, Martin L, Tsai PC, Hamler R, Das AM, Schiffmann R, Johnson FK. Wu YS, et al. Among authors: garcia a. Clin Pharmacol Drug Dev. 2021 Sep;10(9):1075-1088. doi: 10.1002/cpdd.941. Epub 2021 Apr 19. Clin Pharmacol Drug Dev. 2021. PMID: 33876577 Free PMC article.
8,591 results