A look into the future of ALS research

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A review of the current models, including animal models, used in ALS research and new models to accelerate drug discovery.

Although amyotrophic lateral sclerosis (ALS), also referred as ‘Lou Gehrig’s

Disease,’ was first described in 1869 and the first disease-associated gene
was discovered almost 20 years ago, the disease etiology is still not fully
understood and treatment options are limited to one drug approved by the
US Food and Drug Administration (FDA). The slow translational progress
suggests that current research models are not ideal to study such a complicated disease and need to be re-examined. Progress will require greater insight into human genes and biology involved in ALS susceptibility, as well as a deeper understanding of disease phenotype at the histological and molecular levels. Improving human disease outcome will require directing focus toward improved assessment technologies and innovative approaches.

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