News & AnalysisNeurodevelopmental Disorders

New Hope for a Devastating Neurological Disorder

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Science  23 Dec 2011:
Vol. 334, Issue 6063, pp. 1615
DOI: 10.1126/science.334.6063.1615

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People with Angelman syndrome have a nearly normal life span, but the vast majority of them never speak a word. A genetic glitch robs them of speech and leaves them with intellectual disabilities, movement and balance problems, and an excitable demeanor characterized by lots of laughter. Now, scientists have identified a drug that fixes this genetic defect in mice. They warn that it's too early to try the treatment in humans but say they're cautiously optimistic that future therapies may help people with the syndrome lead more normal lives.