新成果发表在日前在线出版的新药《Nature·Medicine》期刊上。新成果发表在日前在线出版的治疗症《自然—医学》期刊上。未来还需要对更大型的肌萎患者小组进行试验。Valentin Gribkoff和同事发现，缩性尽管目前科学家们尚不清楚这种疾病的侧索起因，

参考文献

The 硬化effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis

Merit Cudkowicz, Michael E Bozik, Evan W Ingersoll, Robert Miller, Hiroshi Mitsumoto, Jeremy Shefner, Dan H Moore, David Schoenfeld, James L Mather, Donald Archibald, Mary Sullivan, Craig Amburgey, Juliet Moritz & Valentin K Gribkoff

Amyotrophic lateral sclerosis (ALS) is characterized by upper and lower motor neuron dysfunction and loss, rapidly progressive muscle weakness, wasting and death. Many factors, including mitochondrial dysfunction, may contribute to ALS pathogenesis. Riluzole, which has shown only modest benefits in a measure of survival time without demonstrated effects on muscle strength or function, is the only approved treatment for ALS. We tested the putative mitochondrial modulator dexpramipexole (KNS-760704; (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazole-diamine) in subjects with ALS in a two-part, double-blind safety and tolerability study, with a preliminary assessment of its effects on functional decline and mortality. In part 1, the effects of dexpramipexole (50, 150 or 300 mg d⁻¹) versus placebo were assessed over 12 weeks. In part 2, after a 4-week, single-blind placebo washout, continuing subjects were re-randomized to dexpramipexole at 50 mg d⁻¹or 300 mg d⁻¹as double-blind active treatment for 24 weeks. Dexpramipexole was safe and well tolerated. Trends showing a dose-dependent attenuation of the slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) in part 1 and a statistically significant (P = 0.046) difference between groups in a joint rank test of change from baseline in ALSFRS-R and mortality in part 2 strongly support further testing of dexpramipexole in ALS.

文献链接：https://www.nature.com/nm/journal/v17/n12/full/nm.2579.html

但却无助于缓解肌肉衰弱或功能丧失。新药研究人员发现，治疗症一种名为dexpramipexole的肌萎药物对肌萎缩性脊髓侧索硬化症患者有潜在的治疗效果，导致渐进性的缩性肌肉萎缩和最终的死亡。但线粒体功能性障碍被认为对这种疾病的侧索发展产生了作用。目前的硬化治疗方法只有适度疗效，为了证实这些结果，新药初步迹象显示一种功能尚不知的治疗症药物dexpramipexole能有助于肌萎缩性脊髓侧索硬化症患者临床症状的改善。

在一个小型安慰剂对照试验中，肌萎