ABSTRACT
In progressive disorders such as amyotropic lateral sclerosis (ALS), new therapies can be studied adequately only in the context of randomized controlled trials, which not only determine if a treatment is effective but also increase our overall understanding of the disorder. Rapid innovation at the bench and in trial design have led to a number of recent sophisticated multicenter clinical trials. Yet, despite great strides in both the basic and clinical sciences, progress in ALS research has not so far translated into practical clinical applications. Riluzole remains the only medication approved for use in ALS, nearly a decade after its introduction.
