Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

@article{Mitsumoto2014ClinicalTI,
  title={Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?},
  author={Hiroshi Mitsumoto and Benjamin Rix Brooks and Vincenzo Silani},
  journal={The Lancet Neurology},
  year={2014},
  volume={13},
  pages={1127-1138}
}
Amyotrophic lateral sclerosis (ALS) is one of the most rapidly progressive neurodegenerative diseases of unknown cause. Riluzole is the only drug that slows disease progression. More than 50 randomised controlled trials (RCTs) of proposed disease-modifying drugs have failed to show positive results in the past half-century. In the past decade, at least 18 drugs have been tested in large phase 2 or 3 RCTs, including lithium, which was tested in several RCTs. Potential reasons for the negative… Expand
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