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Published online before print December 16, 2005, doi:10.1212/01.wnl.0000192311.78890.3e)
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NEUROLOGY 2006;66:S110-S113
© 2006 American Academy of Neurology

Treatment and prevention of the amyloidoses

Can the lessons learned be applied to sporadic inclusion-body myositis?

Joel N. Buxbaum, MD

From the Department of Molecular and Experimental Medicine, The Keck Autoimmune Disease Center, Division of Rheumatology Research, The Scripps Research Institute, La Jolla, CA.

Address correspondence and reprint requests to Dr. Joel N. Buxbaum, The Scripps Research Institute, Department of Molecular and Experimental Medicine, The Keck Autoimmune Disease Center, Division of Rheumatology Research, 10550 N. Torrey Pines Road, MEM-230, La Jolla, CA 92037; e-mail: jbux{at}scripps.edu

The amyloid fibril represents a final common pathologic pathway for a variety of human proteins, all of which have a propensity to misfold. Each seems to require a predisposing event to realize its fibrillogenic potential. It may be mutation, inappropriate or incomplete cleavage, overproduction, or the availability of a template for misfolding. Therapies have been based on decreasing the stimulus (inflammation in the case of AA) reducing the number of producing cells (AL) and a variety of approaches to removing the extracellular aggregates. Sporadic inclusion-body myositis (sIBM), while physically resembling the extracellular amyloidoses, is an intracellular disease, hence imposes the additional requirement of developing a therapy that can access and function inside the affected or potentially affected, cell. Current approaches to the treatment of other forms of amyloidosis are discussed in the context of their applicability, or lack thereof, to sIBM.


This article was previously published in electronic format as an Expedited E-Pub at www.neurology.org.

Supported by National Institutes of Health Grant 1R01 AG19259.

Disclosure: The authors report no conflicts of interest.

Received June 22, 2005. Accepted in final form October 14, 2005.




This article has been cited by other articles:


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K. Konstantopoulos, G. Vaiopoulos, A. Mailis, and J. N. Buxbaum
Treatment and prevention of the amyloidoses: Can the lessons learned be applied to sporadic inclusion-body myositis?
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C. E. Finch
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Joel Buxbaum
Neurology Online, 13 Feb 2006 [Full text]
Treatment and prevention of amyloidoses: Can the lessons learned be applied to sporadic IBM?
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Neurology Online, 13 Feb 2006 [Full text]



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