Browsing by Subject "Substrate reduction therapy"
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The design, synthesis and enzymatic evaluation of aminocyclitol inhibitors of glucocerebrosidase
(2014-01-22)Gaucher disease, the most common lysosomal storage disorder, is caused by mutations in the GBA gene which codes for the enzyme glucocerebrosidase (GCase) resulting in its deficiency. GCase deficiency results in the ...
