Publication & Citation Trends
Publications
0 total
Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders OA
Cited by 24
Semantic Scholar
Small molecule inhibition of glycogen synthase 1 restores autophagolysosomal and metabolic pathway dysfunction in a mouse model of Pompe disease
Cited by 0
Semantic Scholar
Quantification of peripheral blood mononuclear cell (PBMC) glycogen as a novel biomarker for therapeutic intervention in Pompe disease
Cited by 0
Semantic Scholar
Substrate reduction therapy for Pompe disease: Small molecule inhibition of glycogen synthase 1 in preclinical models
Cited by 0
Semantic Scholar
Pharmacology of small molecule inhibitors of GYS1 in a mouse model of Pompe disease
Cited by 2
Semantic Scholar
Characterization of glycan substrates accumulating in GM1 Gangliosidosis OA
Cited by 30
Semantic Scholar
Neurofilament light is a treatment‐responsive biomarker in CLN2 disease OA
Cited by 26
Semantic Scholar
Development of an electrochemiluminescent assay to measure LAMP2 in canine cerebrospinal fluid samples
Cited by 0
Semantic Scholar
Research Topics
Lysosomal Storage Disorders Research
(9)
Cancer-related Molecular Pathways
(5)
Carbohydrate Chemistry and Synthesis
(5)
Glycogen Storage Diseases and Myoclonus
(5)
Cellular transport and secretion
(3)
Affiliations
Boston University
Novartis (Switzerland)
Howard Hughes Medical Institute
University of California, San Francisco
Genentech