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52 - Current and potential therapeutics in motor neuron diseases

from Part IX - Motor neuron diseases

Published online by Cambridge University Press:  04 August 2010

M. Flint Beal ,
Anthony E. Lang  and
Albert C. Ludolph
By
Clare Wood-Allum  and
Pamela J. Shaw
M. Flint Beal
Affiliation:
Cornell University, New York
Anthony E. Lang
Affiliation:
University of Toronto
Albert C. Ludolph
Affiliation:
Universität Ulm, Germany
Clare Wood-Allum
Affiliation:
Academic Neurology Unit, Sheffield University Medical School, UK
Pamela J. Shaw
Affiliation:
Academic Neurology Unit, Sheffield University Medical School, UK
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Summary

Introduction

This chapter will survey the research underpinning current therapeutic candidates for amyotrophic lateral sclerosis (ALS), hereditary spastic paraplegia (HSP), Kennedy's disease (SBMA) and spinal muscular atrophy (SMA). It will also review the evidence base and rationale for the use of riluzole in ALS, the only disease-modifying agent currently licensed for use in any neurodegenerative disease of the motor neuron. In the absence of effective therapies to slow disease progression, the focus of management must be on symptomatic therapies aimed at improving quality of life. Guidelines for the symptomatic management of ALS have been generated by the American Academy of Neurology (Miller et al., 1999b) and a number of systematic reviews of the symptomatic management of ALS are also available from the Cochrane library – symptomatic therapies will not be further discussed here (Annane et al., 2000; Langmore et al., 2003). In recent years, improvements in materials science, bioinformatics and the development of exciting new techniques in molecular biology have brought about a revolution in the way that therapeutic candidates are selected and tested. The likely impact of these new techniques on drug development for disorders of motor neurons will also be discussed.

Disease modifying therapies for amyotrophic lateral sclerosis

Aims of therapy

Motor neurons are post-mitotic cells, which make numerous, complex synaptic connections. They are found in the motor cortex, the brainstem and along the entire length of the spinal cord.

Type
Chapter
Information
Neurodegenerative Diseases
Neurobiology, Pathogenesis and Therapeutics
 , pp. 772 - 793
Publisher: Cambridge University Press
Print publication year: 2005

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