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Updated: Jun 11, 2025
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
New developments in the diagnosis and management of motor neuron disease
David O'Brien1, Pamela J Shaw1
1Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield, 285 Glossop Road, Sheffield S10 2HQ, United Kingdom.
Introduction:
Motor neuron disease (MND) is a devastating neurodegenerative disease characterized by progressive muscle weakness.
Sources Of Data:
PubMed, MEDLINE, and Cochrane databases were searched for articles to March 2024. Searches involved the terms 'motor neuron disease' or 'amyotrophic lateral sclerosis' and 'epidemiology', 'diagnosis', 'clinical', 'genetic', 'management', 'treatment', or 'trial'.
Areas Of Agreement:
Evidence-based management involves riluzole, multidisciplinary care, provision of noninvasive ventilation and gastrostomy, and symptomatic treatments. Tofersen should be offered to treat SOD1-MND.
Areas Of Controversy:
Edaravone and Relyvrio are approved treatments in the USA, but insufficient evidence was found to support approval in the UK and Europe.
Growing Points:
The discovery of neurofilaments as MND biomarkers, growth of platform trials and development of novel therapies provide optimism for more powerful neuroprotective therapies.
Areas Timely For Developing Research:
Further work should focus on the elucidation of environmental causes of MND, gene-environment interactions, and advanced cellular models of disease.
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