Recent advances and novel treatments for sphingolipidoses

Volume: 9, Issue: 14, Pages: 1687 - 1700
Published: Sep 1, 2017
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
Volume
9
Issue
14
Pages
1687 - 1700
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