Muscular Dystrophy Specialized Research Center: Project 2

肌营养不良症专业研究中心:项目2

基本信息

  • 批准号:
    10238933
  • 负责人:
  • 金额:
    $ 30.17万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2005
  • 资助国家:
    美国
  • 起止时间:
    2005-06-08 至 2025-06-30
  • 项目状态:
    未结题

项目摘要

Project 2 Summary The long-term goal of Project 2 is to define the natural history of the dystroglycanopathies using established and evolving clinical measures, with the overall objective of optimizing clinical care, and informing and enhancing clinical trial design. This will be accomplished in 3 Aims. In Aim 1, we will extend our current work to define the natural history of FKRP-related dystroglycanopathy. We follow a unique cohort of patients aged early childhood through late adulthood with a range of FKRP mutations. We will expand and extend evaluation of this cohort using standard, validated outcome measures. We will engage those who are no longer able to travel due to disease progression with remote assessments allowing us to relate motor outcomes to real-world endpoints (loss of ambulation, need for respiratory support). We will also add additional evaluation sites for this patient cohort to expand the cohort and confirm that the outcome measures are valid across sites for the FKRP-related patient population. In Aim 2, we will extend our follow up of a cohort of dystroglycanopathy patients with mutations in genes other than FKRP. Each of these genotypes is quite rare. We will identify cohorts that share similar rates of motor progression who might be studied together in gene non-specific clinical trials. We will collect annual blood and urine samples from patients evaluated in Aims 1 and 2 and a subset of these samples will be used in Aim 3 for proteomic biomarker identification. Previous work identified a set of candidate protein biomarkers for patients with FKRP mutations. We will validate these in a separate set of patient samples and determine if any change in concert with disease progression. Discovery of new very low abundance proteins will produce novel candidates for validation.
项目二总结

项目成果

期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)

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Katherine Dianne Mathews其他文献

Katherine Dianne Mathews的其他文献

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{{ truncateString('Katherine Dianne Mathews', 18)}}的其他基金

Component A: Iowa MD STARnet Core Site
组件 A:爱荷华州 MD STARnet 核心站点
  • 批准号:
    10441095
  • 财政年份:
    2019
  • 资助金额:
    $ 30.17万
  • 项目类别:
Component A: Iowa MD STARnet Core Site
组件 A:爱荷华州 MD STARnet 核心站点
  • 批准号:
    10220774
  • 财政年份:
    2019
  • 资助金额:
    $ 30.17万
  • 项目类别:
Component A: Iowa MD STARnet Core Site
组件 A:爱荷华州 MD STARnet 核心站点
  • 批准号:
    10662427
  • 财政年份:
    2019
  • 资助金额:
    $ 30.17万
  • 项目类别:
The University of Iowa's NeuroNEXT Clinical Research Site
爱荷华大学 NeuroNEXT 临床研究中心
  • 批准号:
    10407623
  • 财政年份:
    2018
  • 资助金额:
    $ 30.17万
  • 项目类别:
The University of Iowa's NeuroNEXT Clinical Research Site
爱荷华大学 NeuroNEXT 临床研究中心
  • 批准号:
    10188657
  • 财政年份:
    2018
  • 资助金额:
    $ 30.17万
  • 项目类别:
EVALUATION OF FUKUTIN RELATED PROTEIN MUSCULAR DYSTROPHY
Fukutin相关蛋白性肌营养不良症的评估
  • 批准号:
    7604872
  • 财政年份:
    2007
  • 资助金额:
    $ 30.17万
  • 项目类别:
UNITED DYSTROPHINOPATHY PROJECT
联合肌营养不良项目
  • 批准号:
    7604884
  • 财政年份:
    2007
  • 资助金额:
    $ 30.17万
  • 项目类别:
CLINICAL OUTCOME MEASURES IN FRIEDREICH'S ATAXIA
弗里德赖希共济失调的临床结果测量
  • 批准号:
    7604880
  • 财政年份:
    2007
  • 资助金额:
    $ 30.17万
  • 项目类别:
Muscular Dystrophy Specialized Research Center: Project 2
肌营养不良症专业研究中心:项目2
  • 批准号:
    10442637
  • 财政年份:
    2005
  • 资助金额:
    $ 30.17万
  • 项目类别:
Pathophysiology & therapeutics: Fukutin related protein(FKRP) muscular dystrophy
病理生理学
  • 批准号:
    6960788
  • 财政年份:
    2005
  • 资助金额:
    $ 30.17万
  • 项目类别:

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