Dr. Paul Nyquist points to spots of possible damage caused by a stroke, on a scan of Renee-Nicole Douceur's brain at a news conference at Johns Hopkins Hospital in Baltimore, Friday, Oct. 28, 2011. (AP Photo/Patrick Semansky)
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Scientists have for the first time fixed a protein defect that causes Huntington's disease by injecting a drug from Ionis Pharmaceuticals into the spine, offering new hope for patients with the devastating genetic disease.However, experts cautioned that the results were still early and the ability of the new medicine to improve clinical outcomes for patients had yet to be demonstrated.There is currently no effective disease-modifying treatment for the condition, with existing medicines focused only on managing disease symptoms.The technique has already led to a drug for spinal muscular atrophy that was approved last year.
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