1374752-72-3Relevant academic research and scientific papers
Discovery of a novel noniminosugar acid α glucosidase chaperone series
Xiao, Jingbo,Motabar, Omid,Lea, Wendy A.,Hu, Xin,Zheng, Wei,Southall, Noel,Liu, Ke,Simeonov, Anton,Ferrer, Marc,Marugan, Juan J.,Westbroek, Wendy,Velayati, Arash,Gustafson, Ann Marie,Goldin, Ehud,Sidransky, Ellen,Tamargo, Rafael J.,Ribes, Antonia,Matalonga, Leslie
, p. 7546 - 7559,14 (2020/08/24)
Pompe disease is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the lysosomal enzyme acid α-glucosidase (GAA). Many disease-causing mutated GAA retain enzymatic activity but are not translocated from endoplasmic reticulum
ALPHA-GLUCOSIDASE BINDERS AND METHODS OF THEIR USE
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Page/Page column 36, (2012/05/20)
Compounds that bind to acid alpha glucosidase are described. Methods of using these compounds for the treatment of diabetes and Pompe disease are also described.
