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

COLUMBIA, Mo. Batten disease is a rare, fatal genetic disorder that affects children. Currently, no effective treatment exists for the disease, which ultimately kills all who are affected. Dachshunds also suffer from Batten disease, and now researchers from the University of Missouri College of Veterinary Medicine and School of Medicine, in collaboration with BioMarin Pharmaceutical Inc., have developed a treatment for the disease that has significantly delayed the onset and progression of symptoms in the Dachshunds. The effectiveness of the treatment in the dogs has been so encouraging that plans are underway to initiate human trials of the therapy in children.

Batten disease affects the nervous system in both humans and dogs, causing progressive neural degeneration leading to loss of vision, cognitive and motor function, ability to communicate and, ultimately, death. A number of different forms of Batten disease exist. The treatment developed by MU and BioMarin researchers targets a type of the disease that first becomes evident in the late infantile stage of development. Symptoms for this type of Batten disease begin to appear in patients around the age of two.

Batten disease is caused by the absence of an important enzyme within cells of the neural system. This enzyme helps cells break down and eliminate waste proteins. Without this enzyme, cells accumulate waste and are unable to function properly. Martin Katz, professor of ophthalmology with dual appointments in the MU College of Veterinary Medicine and School of Medicine, along with MU researchers Joan Coates, Fred Wininger, Dennis O'Brien, Gayle Johnson, Jacqueline Pearce and several postdoctoral fellows and students worked with Dachshunds affected with Batten disease in a similar way as humans. For their treatment, a therapeutic protein created by BioMarin Pharmaceuticals Inc., replaces the deficient enzyme and is directly administered into the spinal fluid once every two weeks. Unt
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Contact: Nathan Hurst
hurstn@missouri.edu
573-882-6217
University of Missouri-Columbia
Source:Eurekalert

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