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What Is in the Literature

Bromberg, Mark B. MD, PhD

Journal of Clinical Neuromuscular Disease: December 2019 - Volume 21 - Issue 2 - p 84–89
doi: 10.1097/CND.0000000000000249
Review Article
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This installment of what is in the literature is on amyotrophic lateral sclerosis (ALS). The pathophysiology of ALS remains open and the role of genes, a foothold into pathophysiology, but there are >22 genes identified, and the mechanisms are not known for any. Despite the lack of a firm understanding of pathophysiology, drug trials continue based on possible mechanisms, but no new drugs beyond riluzole and edaravone have been positive in phase 3 trials. There are a number of formal stem cell trials underway, and the results of a phase 2 trial are described. Major efforts to make trials more sensitive are being considered. There are a number of articles with helpful and practical findings for the diagnosis and management of ALS.

Department of Neurology, University of Utah, Salt Lake City, UT.

Reprints: Mark B. Bromberg, MD, PhD, 175 North Medical Drive, Salt Lake City, UT 84132 (e-mail: mark.bromberg@hsc.utah.edu).

The author reports no conflicts of interest.

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