亨廷顿病药物开发:一个第三阶段的管道分析.
Hannah J Van de Roovaart1, Nguyen Nguyen1, Timothy D Veenstra1
1School of Pharmacy, Cedarville University, Cedarville, OH 45314, USA.
Pharmaceuticals (Basel, Switzerland)
|November 25, 2023
概括
本综述总结了亨廷顿病 (HD) 在III期试验中的治疗方法,确定了八种关键药物,包括四种FDA批准的选择,以管理这种神经退行性疾病的症状.
科学领域:
- 神经科学是一个神经科学.
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 亨廷顿病 (HD) 是一种严重的神经退行性疾病,其特点是运动,认知和精神症状.
- 疾病是由自体主导的三核酸重复扩张突变引起的.
- 目前,没有治愈HD的方法,但治疗可以缓解症状.
研究的目的:
- 在亨廷顿病 (HD) 的III期临床试验中识别和总结药物.
- 为提供有关美国当前针对HD的干预治疗方法的资源.
主要方法:
- 对临床试验.gov进行第三阶段的HD干预试验的系统审查.
- 纳入标准:招聘,积极不招聘,或在过去的一年中完成了更新的研究.
- 使用PubMed获取有关确定的试验的补充信息.
主要成果:
- 九项临床试验符合搜索标准.
- 八项试验集中在特定的药物上:甲胺,德克斯特罗梅多芬/丁丁,德特拉贝纳,瓦尔贝纳,Cellavita HD,普罗多匹丁,SAGE-718和RO7234292 (RG6042).
- 调查的四种药物治疗方法已经获得了FDA的批准.
结论:
- 本综述整合了有关目前处于临床开发后期期的有前途的HD治疗方法的信息.
- 突出了FDA批准的治疗方法在III期试验中的可用性.
- 为了解目前亨廷顿病治疗干预的现状提供了宝贵的资源.
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