Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity

Published 2019 View Full Article

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Title
Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity
Authors
Keywords
Sandhoff disease, substrate reduction therapy, glucosylceramide synthase, lysosomal storage disorder, GM2 gangliosidosis, glycosphingolipid, tissue mass spectrometry imaging, in vivo, imaging
Journal
MOLECULAR THERAPY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2019-06-04
DOI
10.1016/j.ymthe.2019.05.018

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