The Evolving Clinical Trial Landscape in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a neurodegenerative disorder affecting predominantly central and peripheral motor neurons, resulting in progressive weakness and atrophy of voluntary skeletal muscle. The disease was originally described by Charcot and Joffroy (1869) but in spite of the extensive scientific knowledge accumulated to date, there is no effective therapeutic strategies. Clinical presentation, diagnostic criteria, traditional and evolving standards for trial design and the increasing importance of including quality of life and healthcare utilisation data in programmes provide the basis for a review.

 
 
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