The glycolipid lysosomal storage diseases are a collection of rare, inherited disorders of metabolism associated with heterogeneous pathologies and reduced life expectancy. Reduction of the substrate that accumulates due to catabolic enzyme deficiency can be mediated by an increasing number of therapeutic approaches, including enzyme replacement, pharmacological intervention to reduce substrate synthesis or enhance residual enzyme activity, and cell or gene therapy. The success of one agent, ...Expand abstract
- Publication status:
- Publisher copy:
- Copyright date:
Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.
If you are the owner of this record, you can report an update to it here: Report update to this record