ARTICLE | Clinical News
uniQure reports preclinical data for Huntington's disease candidate
May 4, 2018 4:29 PM UTC
uniQure N.V. (NASDAQ:QURE) reported preclinical data showing that AMT-130 decreased mutant huntingtin (HTT) protein in animal models of Huntington's disease. Data were presented at the American Academy of Neurology meeting in Los Angeles.
In a pig model of HD, AMT-130 decreased mutant HTT by a median of 68% in the striatum and a median of 47% in the frontal cortex at six months. In a mouse model of HD, AMT-130 improved motor coordination, reduced body weight loss and increased median survival by 24% compared with vehicle controls...
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