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PKU-007: A PHASE 2, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND EFF

PKU-007: A PHASE 2, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND EFF
PKU-007:评估安全性和有效性的 2 期、多中心、开放标签研究
批准号:
7951706
负责人:
Chester B. Whitley
金额:
$0.18万
依托单位:
依托单位国家:
美国
项目类别:
财政年份:
2008
资助国家:
美国
项目状态:
已结题
起止时间:
2008-12-01 至 2009-11-30

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中文摘要
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英文摘要
This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This study will test an experimental medication, Phenoptin, in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency. Hyperphenylalaninemia (HPA) is a very rare disease, estimated to have 450 sufferers worldwide. There is no cure. The mechanism to control this disease is done through strict diet control in order to maintain a low blood phenylalanine level.
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MR Spectroscopy to Determine Neuroinflammation and Oxidative Stress in MPS I (NESTRASIL)
  • 批准号:
    8934179
  • 项目类别:
  • 资助金额:
    $4.56万
  • 财政年份:
    2015
  • 负责人:
    Chester B. Whitley
  • 依托单位:
MR Spectroscopy to Determine Neuroinflammation and Oxidative Stress in MPS I (NESTRASIL)
  • 批准号:
    8907071
  • 项目类别:
  • 资助金额:
    $4.56万
  • 财政年份:
    2014
  • 负责人:
    Chester B. Whitley
  • 依托单位:
The Lysosomal Disease Network's 10th Annual WORLD Symposium
  • 批准号:
    8793924
  • 项目类别:
  • 资助金额:
    $2.0万
  • 财政年份:
    2013
  • 负责人:
    Chester B. Whitley
  • 依托单位:
The Lysosomal Disease Network's 10th Annual WORLD Symposium
  • 批准号:
    8648085
  • 项目类别:
  • 资助金额:
    $1.5万
  • 财政年份:
    2013
  • 负责人:
    Chester B. Whitley
  • 依托单位:
海外基金