Recent advances and novel treatments for sphingolipidoses
Abstract
Sphingolipidoses are genetically inherited diseases in which genetic mutations lead to functional deficiencies in the enzymes needed for lysosomal degradation of sphingolipid substrates. As a consequence, nondegradable lipids enrich in the lysosomes and lead to fatal pathological phenotypes in affected individuals. In this review, different drug-based treatment strategies including enzyme replacement therapy and substrate reduction therapy are...
Paper Details
Title
Recent advances and novel treatments for sphingolipidoses
Published Date
Sep 1, 2017
Journal
Volume
9
Issue
14
Pages
1687 - 1700
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Notes
History