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Brain & Nervous System Health Center

Common Cause of Lou Gehrig's Disease Found

Disease Results From Body's Inability Repair Nerve Cells, Researchers Say
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‘Protein Degradation Pathway’ Key continued...

“Abnormality in protein degradation has been suspected, but there was little direct evidence before this study,” study co-author Han-Xiang Deng, MD, notes in a written statement.

Raymond Roos, MD, the Marjorie and Robert Straus Professor of Neurological Science at the University of Chicago Medical Center, says the discovery appears to represent an important step forward in the understanding of ALS.

He adds that it remains unclear why ubiquilin2 does not function properly in some patients without a family history of ALS.

“This moves the field forward in an impressive way, but like many breakthroughs, many questions remain to be answered,” he says.

There is only one drug approved for the treatment of ALS. It is used to slow disease progression but is not a cure. ALS Association President and CEO Jane H. Gilbert says effective therapies for ALS are long overdue.

“This is very exciting research that has huge promise for the discovery of new treatments,” she tells WebMD.

Lucie Bruijn, PhD, scientific director of the ALS Association, says she is optimistic that better drugs or drug combinations will emerge to slow disease progression.

Siddique is also optimistic that his team’s discovery could lead to better treatments for ALS and other diseases of the nerves.

“This is the most hopeful I have been in 25 years of research,” he says. “Previously, we were running in many different directions, but this is where we will focus from now on.”

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