Idebenone in patients with Friedreich ataxia
Idebenone in patients with Friedreich ataxia
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DOI：
10.1016/s0304-3940(01)01892-4
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发表时间：
2001-06-29
影响因子：
2.5
通讯作者：
Zange, J
中科院分区：
文献类型：
--
作者：
Schöls, L;Vorgerd, I;Zange, J
Friedreich共济失调(Friedreich ataxia，FA)是最常见的退行性共济失调，被认为是由于线粒体铁积累和氧化应激引起的呼吸/缺乏引起的。自由基清除剂艾地苯酮对FA体外模型线粒体功能有保护作用。在一项安慰剂对照的交叉试验中，我们研究了艾地苯酮对9名非卧床FA患者呼吸功能的影响。P-31磁共振波谱显示，所有FA患者的骨骼肌线粒体均有损伤，但依地苯酮治疗后未见恢复。临床疗效不明显。超声心动图没有证实关于艾地苯酮改善FA相关心肌病的初步研究。(C)2001爱思唯尔爱尔兰科学有限公司。保留所有权利。
Friedreich ataxia (FA), the most common form of degenerative ataxia, is thought to be caused by respiraton/deficiency due to mitochondrial iron accumulation and oxidative stress. Idebenone, a free-radical scavenger, protects mitochondrial function in in vitro models of FA. In a placebo-controlled crossover trial we studied the effect of idebenone on respiratory function in nine ambulant FA patients. P-31 magnetic resonance spectroscopy demonstrated mitochondrial impairment in vivo in skeletal muscle of all FA patients, but no recovery with idebenone. No effects were seen in clinical sco res. Echocardiography did not confirm a preliminary study reporting improvement of FA-associated cardiomyopathy with idebenone. (C) 2001 Elsevier Science Ireland Ltd. All rights reserved.