Journal
NEUROTHERAPEUTICS
Volume 5, Issue 4, Pages 516-527Publisher
SPRINGER
DOI: 10.1016/j.nurt.2008.08.002
Keywords
amyotrophic lateral sclerosis; clinical trial; trial design; conduct; critical review
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Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
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