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Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis.

Publication ,  Journal Article
Bedlack, RS; Traynor, BJ; Cudkowicz, ME
Published in: Expert Opin Emerg Drugs
May 2007

It has been > 130 years since the first description of the upper and lower motor neuron disease called amyotropic lateral sclerosis (ALS). Sadly, there has been little change in the long interval over which this disease is diagnosed, or in its poor prognosis. Significant gains have been made, however, in understanding its pathophysiology and in symptomatic care. Disease-causing mutations have been identified and used to create animal models. Other identified mutations may increase susceptibility and cause disease only in a particular environment and at a particular age. A number of 'downstream' molecular pathways have been implicated, including transcriptional disturbances, protein aggregation, excitotoxicity, mitochondrial dysfunction, oxidative stress, neuroinflammation, cytoskeletal and axonal transport derangements, growth factor dysregulation and apoptosis. This knowledge has led to an impressive pipeline of candidate therapies that offer hope for finally being able to alter ALS disease progression. These are described and prioritized herein, and suggestions are offered for efficiently sifting through them.

Duke Scholars

Published In

Expert Opin Emerg Drugs

DOI

EISSN

1744-7623

Publication Date

May 2007

Volume

12

Issue

2

Start / End Page

229 / 252

Location

England

Related Subject Headings

  • Signal Transduction
  • Pharmacology & Pharmacy
  • Neuroprotective Agents
  • Motor Neuron Disease
  • Humans
  • Animals
  • Amyotrophic Lateral Sclerosis
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

APA
Chicago
ICMJE
MLA
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Bedlack, R. S., Traynor, B. J., & Cudkowicz, M. E. (2007). Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis. Expert Opin Emerg Drugs, 12(2), 229–252. https://doi.org/10.1517/14728214.12.2.229
Bedlack, Richard S., Bryan J. Traynor, and Merit E. Cudkowicz. “Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis.Expert Opin Emerg Drugs 12, no. 2 (May 2007): 229–52. https://doi.org/10.1517/14728214.12.2.229.
Bedlack RS, Traynor BJ, Cudkowicz ME. Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis. Expert Opin Emerg Drugs. 2007 May;12(2):229–52.
Bedlack, Richard S., et al. “Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis.Expert Opin Emerg Drugs, vol. 12, no. 2, May 2007, pp. 229–52. Pubmed, doi:10.1517/14728214.12.2.229.
Bedlack RS, Traynor BJ, Cudkowicz ME. Emerging disease-modifying therapies for the treatment of motor neuron disease/amyotropic lateral sclerosis. Expert Opin Emerg Drugs. 2007 May;12(2):229–252.

Published In

Expert Opin Emerg Drugs

DOI

EISSN

1744-7623

Publication Date

May 2007

Volume

12

Issue

2

Start / End Page

229 / 252

Location

England

Related Subject Headings

  • Signal Transduction
  • Pharmacology & Pharmacy
  • Neuroprotective Agents
  • Motor Neuron Disease
  • Humans
  • Animals
  • Amyotrophic Lateral Sclerosis
  • 3214 Pharmacology and pharmaceutical sciences
  • 1115 Pharmacology and Pharmaceutical Sciences