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Therapy Yields Promise for Fatal Neurological Condition
Date:5/13/2008

sease, but today, we can say that there is some hope," Crystal said. "These results are not just promising for sufferers of the disease, but suggest that gene therapy can work and should be studied for other neurological disorders. Each gene in our body has the potential to become a target to study for human disease."

Only about 200 cases of Batten disease exist in the world at a given time because of its early fatality rate. Around age 4, children with Batten disease usually start showing symptoms such as impaired muscle coordination (ataxia), involuntary twitching (myoclonus), and speech and developmental disorders. Eyesight then usually begins to decline, and affected children generally become wheelchair-bound, then bedridden.

More information

The National Institute of Neurological Disorders and Stroke has more about Batten disease.



-- Kevin McKeever



SOURCE: NewYork-Presbyterian Hospital/Weill Cornell Medical Center, news release, May 13, 2008


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