...
  • 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>The EPMA journal. >Recent advances in amyotrophic lateral sclerosis research: perspectives for personalized clinical application
【24h】

Recent advances in amyotrophic lateral sclerosis research: perspectives for personalized clinical application

机译：肌萎缩性侧索硬化研究的最新进展：个性化临床应用的前景

获取原文
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Treatment of amyotrophic lateral sclerosis (ALS) has been fueled, in part, by frustration over the shortcomings of the symptomatic drugs available, since these do not impede the progression of this disease. Currently, over 150 different potential therapeutic agents or strategies have been tested in preclinical models of ALS. Unfortunately, therapeutic modifiers of murine ALS have failed to be successfully translated into strategies for patients, probably because of differences in pharmacokinetics of the therapeutic agents, route of delivery, inefficiency of the agents to affect the distinct pathologies of the disease or inherent limitations of the available animal models. Given the multiplicity of the pathological mechanisms implicated in ALS, new therapies should consider the simultaneous manipulation of multiple targets. Additionally, a better management of ALS therapy should include understanding the interactions between potential risk factors, biomarkers and heterogeneous clinical features of the patients, aiming to manage their adverse events or personalize the safety profile of these agents. This review will discuss novel pharmacological approaches concerning adjusted therapy for ALS patients: iron-binding brain permeable multimodal compounds, genetic manipulation and cell-based treatment.
机译：肌萎缩性侧索硬化症（ALS）的治疗部分是由于对现有症状药物的不足感到沮丧，因为这些药物不会阻碍该病的进展。目前，已经在ALS的临床前模型中测试了150多种不同的潜在治疗剂或策略。不幸的是，鼠ALS的治疗修饰剂未能成功地转化为患者的治疗策略，这可能是由于治疗剂的药代动力学差异，递送途径，影响疾病独特病理的药物无效或固有的局限性所致。可用的动物模型。鉴于涉及ALS的多种病理机制，新疗法应考虑同时操纵多个靶标。此外，更好的ALS治疗管理应包括了解潜在危险因素，生物标记物和患者异质临床特征之间的相互作用，以管理其不良事件或个性化这些药物的安全性。这篇综述将讨论有关ALS患者调整疗法的新药理学方法：铁结合的脑可渗透多式联运化合物，基因操作和基于细胞的治疗。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号