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New treatment targets found for ALS

PHILADELPHIA, June 18 (UPI) -- U.S. scientists have discovered misfolded proteins called TDP-43 accumulate throughout the brains of patients suffering amyotropic lateral sclerosis, or ALS.

Previous research at the University of Pennsylvania had shown TDP-43 accumulated in the motor areas of the brains of people with ALS, also known as Lou Gehrig's disease. Now the same researchers have shown TDP-43 actually accumulates throughout the brain, suggesting ALS has broader neurological effects than previously appreciated.

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"The primary implication for ALS patients is that we have identified a molecular target for new therapies," said study co-author Dr. John Trojanowski, director of the university's Institute on Aging. "The other implication is that new therapies for ALS now need to go beyond treating only motor neurons.

"Our observation of TDP-43 in the brains of ALS patients suggests that ALS and two other neurodegenerative diseases called ALS-plus (ALS with cognitive impairments) and FTLD (frontotemporal lobar disease) may all have the same underlying molecular pathology involving abnormal TDP-43," added Trojanowski. "This constitutes a paradigm shift in the way we think about these diseases."

The study appeared in last month's issue of the Archives of Neurology.

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