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Amyotrophic Lateral Sclerosis

Findings suggest current approach to drug discovery for Lou Gehrig's disease be re-examined

Published in Drug Week, May 25th, 2007

Most research on Lou Gehrig’s disease therapeutics has been based on the assumption that its two forms (sporadic and hereditary) are similar in their underlying cause. Now, researchers at the University of Pennsylvania School of Medicine have found an absolute biochemical distinction between these two disease variants, suggesting that current approaches to drug discovery should be re-examined.

About 5 percent of all cases of Lou Gehrig’s disease, or amyothrophic lateral sclerosis (ALS), are passed from generation to generation. The most common genetic variant in this familial form is caused by a mutation in the SOD-1 gene. The researchers looked at a large set of ALS...

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