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中文摘要
翻译
帕金森病(PD)是一种缓慢但无情的进行性神经退行性疾病,导致 临床症状呈时间依赖性恶化。抗帕金森病药物能有效改善大部分主要疾病。 大多数患者出现帕金森氏症的体征和症状。然而,传统的药物疗法不起作用。 随着时间的推移,运动功能的进行性恶化和功能残疾的恶化。因此, 疾病修改药物,可以减缓临床进展,促进受损神经元的修复,恢复或 增强多巴胺(DA)系统残留部分的功能是非常必要的。自病以来 进展通常是缓慢的,使用针对多种致病机制的化合物治疗可能是 在相对较长的时间内实施。NET-PD多中心试验旨在测试 潜在神经保护化合物在早期帕金森病患者中的疗效。这些化合物是 在无效性研究中被确定有效的将在大型双盲安慰剂对照中进行测试 学习。NINDS的神经退行性变和临床试验小组正在申请新的 中心合作进行临床试验,以测试潜在的神经保护剂。我们正在提议成为 Net-PD中心。肯塔基大学的运动障碍中心为大列克星敦 区域,主要是城市社区,以及中部、东部和东南部的63个县的主要农村地区 肯塔基州。此外,我们还治疗居住在西弗吉尼亚州和俄亥俄州南部的几名PD患者(-25岁)。 我们跟踪了600名PD患者,平均每个月诊断出5名新的PD患者。因此,我们 预计每月能够招募1-2名早期PD患者参加试点试验。
英文摘要
Parkinson's disease (PD) is a slowly but relentlessly progressive neurodegenerative disorder resulting in a time-dependent worsening of clinical symptoms. Anti-PD drugs effectively ameliorate most of the major parkinsonian signs and symptoms in a majority of patients. However, traditional drug therapies do not modify the progressive deterioration of motor function and functional disability worsens over time. Thus, disease modifying drugs that can slow clinical progression, enhance repair of damaged neurons, restore or enhance function of residual parts of the dopamine (DA) system are much needed. Since disease progression is generally slow, treatment with compounds that target multiple pathogenic mechanisms can be implemented over a relatively long period of time.The NET-PD multicenter trial is aimed at testing the efficacy of potential neuroprotective compounds in early stage PD patients. Compounds that are ascertained to be effective in the Futility studies will be tested in large double-blinded placebo control studies. The Neurodegeneration and Clinical Trial Groups of the NINDS are requesting applications for new centers to collaborate in clinical trials to test potential neuroprotective agents. We are proposing to become a NET-PD center. The Movement Disorders Center at University of Kentucky serves the greater Lexington area, a primarily urban community and the largely rural, 63-county area of central, eastern, and southeastern Kentucky. In addition, we treat several PD patients (-25) who reside in West Virginia and Southern Ohio. We follow -600 PD patients and we diagnose on average 5 new PD patients per month. Therefore, we expect to be able to enroll 1-2 early stage PD patients per month into the Pilot Trials.
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Cognitive decline in aging and AD: neuroprotection by hypermyelination in FusOLcKO
Novel ALS models: FUS conditional knockout in motor neurons and oligodendrocytes
  • 批准号:
    9026836
  • 项目类别:
  • 资助金额:
    $0.0万
  • 财政年份:
    2016
  • 负责人:
    FRANCA CAMBI
  • 依托单位:
国内基金
海外基金
Molecular Interaction Reconstruction of Rheumatoid Arthritis Therapies Using Clinical Data