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JAMA Neurol:氨溴索联合β-葡萄糖脑苷酶用于改善PD患者运动功能

2020-01-19 MedSci MedSci原创

研究发现,氨溴索治疗安全、耐受性好,能穿透血脑屏障,实现靶向结合,提高脑脊液α-突触核蛋白水平,改善帕金森患者运动功能

葡萄糖脑糖苷酶基因GBA1(OMIM 606463)突变是帕金森病(PD)最重要的危险因素。体外和体内研究表明,氨溴索可提高β-葡萄糖脑苷酶(GCase)酶活性,降低α-突触核蛋白水平。近日研究人员探讨了盐酸氨溴索联合GCase治疗PD伴和不伴GBA1基因突变患者的安全性、耐受性、脑脊液(CSF)渗透性及靶向性。

23例中度PD患者参与研究,18名患者完成,每天口服氨溴索的剂量增加到1.26克。研究的主要终点为186天脑脊液中氨溴索检出量和脑脊液GCase活性变化。

18名参与者中,15名为男性,平均年龄60.2岁,17人(8人有GBA1突变,9人没有GBA1突变)被纳入初步分析。在第0天到第186天之间,观察到脑脊液中氨溴索含量增加了156 ng/mL。脑脊液GCase活性下降19%(0.059nmol/mL/h)。氨溴索治疗耐受性良好,无严重不良反应。脑脊液α-突触核蛋白浓度增加50pg/mL(13%)。脑脊液GCase蛋白质水平增加88ng/mol(35%)。帕金森病统一运动障碍评分下降(即改善)6.8分。在有和无GBA1突变的患者中均观察到上述变化。

研究发现,氨溴索治疗安全、耐受性好,能穿透血脑屏障,实现靶向结合,提高脑脊液α-突触核蛋白水平,改善帕金森患者运动功能。

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    2020-06-04 yinhl1978
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    2020-01-19 旺医

    顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息

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