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Expert Opin Investig Drugs. 2007 Aug;16(8):1197-207.

Current clinical trials in amyotrophic lateral sclerosis.

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Columbia University, Eleanor and Lou Gehrig MDA/ALS Research Center, Department of Neurology, Neurological Institute, New York, NY 10032, USA.


Amyotrophic lateral sclerosis is caused by selective degeneration of motor neurons in the brain and spinal cord. There are still no other effective therapies 10 years after the approval of riluzole for the treatment of amyotrophic lateral sclerosis, but advances in drug development and screening are substantially increasing the number of potential therapeutic agents. This review provides an overview of clinical trial methodology in amyotrophic lateral sclerosis followed by a systematic evaluation of drugs that are presently in Phase I, II and III clinical trials. There is an emphasis on the scientific evidence supporting the selection of each drug being tested, as well as on trial design.

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