File:Dddt-7-729Fig1.jpg
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Summary
Description |
English: Sulfatide metabolic pathway.
Notes: The figure shows the biosynthetic pathways of sulfatide, the sphingolipid that accumulates during metachromatic leukodystrophy MLD. The specific enzymes described in each step of the pathway are in italics. The rounded boxes depict the products or substrates of the reactions. The green shaded square shapes show different therapeutic approaches for MLD treatment. Two current substrate reduction therapies for another lysosomal disease (Gaucher disease) are also shown: N-Butyldeoxynojirimycin (NB-DNJ) miglustat (Zavesca®, Actelion Pharmaceuticals Ltd, Allschwil, Switzerland), and eliglustat tartrate. Both of these small molecules competitively inhibit ceramide glucosyltransferase. Abbreviations: ASA, arylsulfatase A; EET, enzyme enhancement therapy; ERT, enzyme replacement therapy; Gal, galactose; HSCT, hematopoietic stem cell transplantation; Glc, glucose; SGal-Glc-cer, sulfo-galactose-glucosylceramide; MLD, metachromatic leukodystrophy; SRT, substrate reduction therapy. |
Date | |
Source | https://pubmed.ncbi.nlm.nih.gov/23966770/ |
Author | Shilpa A Patil , Gustavo H B Maegawa |
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