Harnessing polyhydroxylated pyrrolidines as a stabilizer of acid alpha-glucosidase (GAA) to enhance the efficacy of enzyme replacement therapy in Pompe disease

Li, HY; Lee, NC; Chiu, YT; Chang, YW; Lin, CC; Chou, CL; Chien, YH; Hwu, WL; Cheng, WC

Cheng, WC (通讯作者)，Acad Sinica, Genom Res Ctr, 128,Acad Rd,Sect 2, Taipei 11529, Taiwan.;Hwu, WL (通讯作者)，Natl Taiwan Univ Hosp, Dept Pediat & Med Genet, 8 Chung Shan South Rd, Taipei 10041, Taiwan.

BIOORGANIC & MEDICINAL CHEMISTRY, 2023; 78 ():

Abstract

To discover small molecules as acid alpha-glucosidase (GAA) stabilizers for potential benefits of the exogenous enzyme treatment toward Pompe disease ......

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