Gene AMT-130疗法(AMT-130)在一项小型研究中将亨廷顿的进度减慢了高达75%,但结果需要更大规模的试验才能证实。 Gene therapy AMT-130 slowed Huntington’s progression by up to 75% in a small study, but results need larger trials for confirmation.

一项名为AMT-130的基因疗法在对29名病人进行的未经审查的小型研究中将亨廷顿病情发展速度减慢了75%,36个月来,接受高剂量治疗的人受益最大。 A gene therapy called AMT-130 slowed Huntington’s disease progression by up to 75% in a small, unreviewed study of 29 patients, with higher-dose recipients showing the most benefit over 36 months. 通过脑外科手术,治疗降低了脊椎液中的有害的狩猎蛋白质水平和神经损伤标记,改善了运动、认知和日常功能。 Delivered via brain surgery, the treatment reduced harmful huntingtin protein levels and nerve damage markers in spinal fluid, with improvements in movement, cognition, and daily function. 虽然耐受性很好,但副作用与侵入性手术有关. While well-tolerated, side effects were linked to the invasive procedure. 专家们告诫说,结果应是初步的,以外部比较为基础,而不是以有控制的试验为基础,并需要通过更大的研究加以确认,然后才能得到监管批准,而uniQure计划在2026年寻求批准。 Experts caution that results are preliminary, based on external comparisons rather than a controlled trial, and require confirmation through larger studies before regulatory approval, which uniQure plans to seek in 2026.