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Annals of Translational Medicine|August 9, 2019
Challenges in treating Pompe disease: an industry perspectiveHung V Do, Richie Khanna, Russell GotschallThe Journal of Biological Chemistry|May 10, 2021
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidaseNithya Selvan, Nickita Mehta, Suresh Venkateswaran, et al.Human Mutation|October 29, 2009
The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidaseJohn J Flanagan, Barbara Rossi, Katherine Tang, et al.Plos One|July 19, 2014
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 diseaseRichie Khanna, Allan C Powe, Yi Lun, et al.JCI Insight|March 8, 2019
Improved efficacy of a next-generation ERT in murine Pompe diseaseSu Xu, Yi Lun, Michelle Frascella, et al.Pageof 1