Authors: Moore, David F; Brady, Roscoe O

Source: Future Lipidology, Volume 3, Number 1, February 2008 , pp. 105-111(7)

Publisher: Future Medicine

Abstract:

The discovery of the enzymatic defects in sphingolipid-storage disorders led to important benefits for patients with these conditions. Diagnostic tests, genetic counseling procedures and prenatal diagnosis are available for these disorders. Enzyme-replacement therapy is especially effective for patients with Gaucher disease. It is also approved for patients with Fabry disease, but as yet unrecognized targets need to be addressed and treatment improved, probably because of the multiplicity of organs and tissues involved in patients with this condition. We propose that a significant increase of our understanding of the pathophysiology and etiopathogenesis underlying Fabry disease and other metabolic storage disorders is probably required to develop effective therapies for these patients. Significant advances in this aspect of Fabry disease and other disorders are anticipated from the application of a system- biology approach to understand the cellular and metabolic consequences of lipid accumulation. This strategy is discussed and its potential evaluated in this overview.

Keywords: hereditary metabolic storage disorders; improved therapeutic strategies; sphingolipidoses; systems biology

Document Type: Research article

DOI: http://dx.doi.org/10.2217/17460875.3.1.105

Publication date: 2008-02-01

More about this publication?

• Future Lipidology addresses therapeutic strategies and emerging topics in this complex area of cardiovascular medicine. The journal delivers essential information in concise, at-a-glance article formats. Key advances in the field are reported and analyzed by international experts, providing an authoritative but accessible forum.
• Editorial Board
• Information for Authors
• Submit a Paper
• Subscribe to this Title
• Information for Advertisers
• Terms & Conditions
• Information for Librarians
• E-Access Trials
• ingentaconnect is not responsible for the content or availability of external websites

Related content

• In this: publication
• By this: publisher
• In this Subject: Medicine
• By this author: Moore, David F ;  Brady, Roscoe O

Website © Publishing Technology. Article copyright remains with the publisher, society or author(s) as specified within the article.

• Terms and conditions
• Privacy policy
• Information for advertisers