Skip to main content Accessibility help

We use cookies to distinguish you from other users and to provide you with a better experience on our websites. Close this message to accept cookies or find out how to manage your cookie settings.

Close cookie message
×
  1. Home
  2. Search

Refine search

Refine search

Access:
Content type:
Publication date:
Apply   From and To filters
Subject: Show more
Journals Show more
Publishers: Show more
Societies Show more
Series: Show more
Collections: Show more

Actions for selected content:

Select all | Deselect all
  • View selected items
  • Export citations
  • Download PDF (zip)
  • Save to Kindle
  • Save to Dropbox
  • Save to Google Drive

Save Search

You can save your searches here and later view and run them again in "My saved searches".

Please provide a title, maximum of 40 characters.
Cancel
×

1 results

  • Chapter 20 - Promises and challenges of translational research in neuropsychiatry

  • Edited by James E. Barrett, Joseph T. Coyle, Michael Williams
  • Book:
    Translational Neuroscience
    Published online:
    05 July 2012
    Print publication:
    28 June 2012, pp 339-358

  • Summary

    Amyotrophic lateral sclerosis (ALS) is the most common form of adult motor neuron disease. A brief overview of some of the more important genes identified in familial ALS is provided. To date, riluzole is the only drug that has demonstrated efficacy in patients with ALS. One of the most challenging aspects in translating candidate therapeutic agents for ALS to clinical use is the appropriate clinical trial design. ALS-relevant compounds has recently increased, and the potential for interfering RNA (RNAi) and antisense technologies to target a specific but well-characterized (mutant superoxide dismutase 1) protein responsible for many cases of familial ALS may offer proof of the principle that ALS can be effectively treated if the upstream causative etiologies are identified. Finally, recent advances in stem cell research, including the development of induced pluripotent stem cells, offer the potential for autologous cell transplantation as well as drug discovery.