55. An adjunct rhGAA ERT-small molecule treatment approach for Pompe disease: Pharmacological chaperone AT2220 prevents rhGAA denaturation and loss of enzyme activity
Titel:
55. An adjunct rhGAA ERT-small molecule treatment approach for Pompe disease: Pharmacological chaperone AT2220 prevents rhGAA denaturation and loss of enzyme activity
Auteur:
Flanagan, John J. Khanna, Richie Neusch, George Feng, Jessie Zdancewicz, Michelle Ranes, Brian Soska, Rebecca Yin, Terry Valenzano, Ken J. Lockhart, David J. Do, Hung V.