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Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.

Abstract:

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, ...

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Publication status:
Published

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Publisher copy:
10.1517/14656566.8.4.427

Authors


Butters, TD More by this author
Journal:
Expert opinion on pharmacotherapy
Volume:
8
Issue:
4
Pages:
427-435
Publication date:
2007-03-05
DOI:
EISSN:
1744-7666
ISSN:
1465-6566
URN:
uuid:645c8136-f069-4403-bd6a-e145eeea9820
Source identifiers:
101049
Local pid:
pubs:101049

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