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美国麻省总医院神经学研究和临床试验小组的Cudkowicz等人于近期报道了一项临床研究结果,称由BiogenIdec公司和Knopp Neurosciences公司合作开发的小分子线粒体功能调节剂dexpramipexole(代号:KNS-760704)或可治疗肌萎缩性脊髓侧索硬化症(amyotrophic lateral sclerosis,ALS)。该项由ALS患者参加的研究由PART1和PART2两部分组成,其中,PART1研究对dexpramipexole(50、150和300 mg.d-1)与安慰剂治疗12周时的疗效进行
Cudkowicz et al. Recently reported the results of a clinical study published by the Massachusetts General Hospital Neurology Research and Clinical Trials Group that dexpramipexole (code: KNS-760704), a small molecule mitochondrial function regulator developed by BiogenIdec and Knopp Neurosciences ) Or treatable amyotrophic lateral sclerosis (ALS). This ALS-participant study consisted of PART1 and PART2, where the PART1 study evaluated the efficacy of dexpramipexole (50, 150 and 300 mg.d-1) at placebo for 12 weeks