对唐氏综合征成年人粉样向抗体的处方标准的调整
Hampus Hillerstrom1, Richard Fisher1, Matthew P Janicki2,3
1LuMind IDSC Foundation, Burlington, Massachusetts, USA.
概括
患有唐氏综合征 (DS) 的成年人因年龄和诊断标准而面临进入阿尔茨海默氏症治疗的障碍. 专家建议适应早期痴呆症发病的标准,促进公平获得免疫治疗药物.
科学领域:
- 神经学 神经学
- 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:
- 目前FDA批准的抗粉样蛋白免疫治疗药物针对晚发性阿尔茨海默病 (AD).
- 国家表格标准排除了患有唐氏综合征 (DS) 的成年人,因为痴呆发病和诊断概况不同.
- 这种排除限制了成年DS患者获得可能改变疾病的治疗方法的机会.
研究的目的:
- 为解决与唐氏综合征患者成年人获得抗粉样蛋白免疫疗法的差异.
- 建议对DS患者的处方标准和准入参数进行调整.
- 促进DS和认知衰退患者在医疗保健中的公平.
主要方法:
- 召集了一个国际专家小组.
- 为DS患者制定了适应的处方标准.
- 建议对医疗保险和医疗补助服务中心 (CMS) 和付款人标准进行修改.
- 推用于DS认知衰退的验证的替代评估工具.
- 启动了提高意识的活动,并与联邦官员进行了接触.
主要成果:
- 拟议的修改是为了考虑到DS的较年轻发病年龄.
- 建议使用验证的替代语言和DS的评估工具.
- 增强临床医生对DS相关认知衰退的诊断能力.
- 协助与联邦官员就公平获取问题进行讨论.
结论:
- 适应当前的标准对于成年患有DS的人平等获得抗粉样蛋白疗法至关重要.
- 解决诊断和与年龄有关的差异可以减轻医疗保健不平等.
- 这些适应对全球监管机构产生影响,这些机构评估阿尔茨海默病的新疗法.
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