基因疗法使亨廷顿病情在临床试验3年中减缓75%, A gene therapy slowed Huntington’s disease progression by 75% over 3 years in a clinical trial, marking the first disease-modifying treatment for the inherited disorder.

一种叫做AMT-130的基因疗法在临床试验中首次减缓了亨廷顿病情的发展速度,表明接受高剂量治疗的病人在36个月中下降了75%。 A gene therapy called AMT-130 has slowed Huntington’s disease progression for the first time in a clinical trial, showing a 75% reduction in decline over 36 months for patients receiving a high dose. 由uniQure开发并由伦敦大学学院测试,一次性脑外科治疗改善了或稳定了运动、认知和功能能力,脑损伤标记水平较低。 Developed by uniQure and tested by University College London, the one-time brain surgery treatment improved or stabilized motor, cognitive, and functional abilities, with lower levels of a brain injury marker. 研究人员称结果具有变革性,指出有些病人保持稳定或恢复工作。 Researchers called the results transformative, noting some patients remained stable or returned to work. 通过单一剂量提供的治疗预计将带来长期的好处,并代表了对这种遗传的神经退化性疾病进行疾病改变治疗的第一批证据。 The therapy, delivered via a single dose, is expected to offer long-lasting benefits and represents the first evidence of a disease-modifying treatment for this inherited neurodegenerative disorder.