对儿童耐药性的药物治疗策略
Stéphane Auvin1, Nicola Specchio2
1APHP, Robert Debré University Hospital, Pediatric Neurology Department, CRMR epilepsies rares, EpiCare member, Paris, France; Université Paris Cité, INSERM NeuroDiderot, Paris, France; Institut Universitaire de France, (IUF), Paris, France.
的耐药性意味着药物无法阻止发作. 排除其他原因和探索精准医学或基因疗法可能为治疗提供新的希望.
科学领域:
- 神经学 神经学
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 药物耐药性是管理中的一个重大挑战,由多种抗发作药物的失败来定义.
- 在儿科病例中,必须排除伪耐药性,包括不遵守或误诊.
- 尽管取得了进展,但耐药性的发病率仍然保持不变.
研究的目的:
- 讨论抗发作药物的选择.
- 探索多疗法和精准医学在治疗中的概念.
- 审查的新兴基因疗法.
主要方法:
- 关于中药物耐药性的文献评论.
- 讨论治疗策略,包括多疗法和精准医学.
- 基因治疗方法的概述,如反意义寡核酸和AAV载体.
主要成果:
- 选择抗发作药物需要仔细考虑个体患者的因素.
- 精准医学为罕见的机制驱动治疗提供了潜力.
- 基因疗法正在向针对特定综合征的人体试验取得进展.
结论:
- 在耐药性中,当手术不是一种选择时,治疗目标转向改善生活质量.
- 精准医学在抗药性的疗效需要进一步研究.
- 基因疗法有望改变某些类型的过程.
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