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Effective treatment for late infantile batten disease developed by MU, BioMarin researchers
Date:2/11/2013

reated dogs ultimately succumbed to the disease around 10 to 11 months of age. Dogs treated with the enzyme replacement therapy showed significant delays in the onset of symptoms and survived ! substantially longer.

"This is an important step toward treating human patients with this debilitating disease," Katz said. "By introducing a replacement for the missing enzyme into the nervous system, we have been able to help the deficient cells eliminate their waste efficiently and slow the disease-related brain degeneration. We believe this treatment approach will be effective in humans as well. Based on research to date, this treatment does not appear to result in a complete cure for the disease, but it could extend the lives and improve the quality of life for those with this form of Batten disease."

"The researchers at MU have painstakingly characterized Late-Infantile Batten disease in these dogs, and their results indicate a striking similarity in the progression of the disease among dogs and humans," said Charles O'Neill, vice president of pharmacological sciences at BioMarin. "Treatment of the dogs with BioMarin's enzyme replacement therapy has characterized its safety and efficacy, and will enable accelerated clinical development of this potential treatment for this devastating disease."


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Contact: Nathan Hurst
hurstn@missouri.edu
573-882-6217
University of Missouri-Columbia
Source:Eurekalert

Page: 1 2

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