丁苯酞治疗神经系统变性病及遗传性疾病的疗效观察

Therapeutic Effects of D1-3n-Butyphthalide on Patients with Neurodegenerative and Neurogenetic Diseases

目的初步评价丁苯酞对神经系统变性病及遗传性疾病的临床治疗效果。方法4例神经系统变性病及遗传性疾病患者均给予丁苯酞治疗6个月,分别于治疗4周、8周、6个月后观察其疗效并对其进行分析。结果4例患者在服用丁苯酞6个月后2例显效、2例有效。结论丁苯酞通过保护和改善线粒体功能有望成为神经系统变性病及遗传疾病的有效治疗手段,但尚需大样本临床试验证实。

Objective To preliminarily evaluate the therapeutic effects of d1-3n-butyph- thalide on patients with neurodegenerative and neurogenetic diseases. Methods Four patients with neurodegenerative and neurogenetic diseases were treated by dl-3n-butyphthalide for 6 months and the therapeutic effects were observed and analyzed 4 weeks,8 weeks and 6 months after oral administration. Results Two cases were found with significant effectiveness and the other 2 cases had slight effectiveness. Conclusion Dl-3n-butyphthalide may be effective in treating neurodegenerative and neurogenetic diseases due to its role in protecting and improving the function of mitochondria. Yet large-scale methodologically-sound trials are still required.