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
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
关键词:
BloodClinical ResearchComputer Retrieval of Information on Scientific Projects DatabaseDietFundingGrantHyperphenylalaninaemiasInstitutionPharmaceutical PreparationsPhasePhenylalanineRare DiseasesResearchResearch PersonnelResourcesSafetySourceTestingUnited States National Institutes of Healthdisorder controlopen labelphenoptintetrahydrobiopterin
中文摘要
点击翻译按钮获取中文摘要
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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会议论文
MR Spectroscopy to Determine Neuroinflammation and Oxidative Stress in MPS I (NESTRASIL)
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批准号:8934179
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项目类别:
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资助金额:$4.56万
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财政年份:2015
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负责人:Chester B. Whitley
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依托单位:
MR Spectroscopy to Determine Neuroinflammation and Oxidative Stress in MPS I (NESTRASIL)
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批准号:8907071
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项目类别:
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资助金额:$4.56万
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财政年份:2014
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负责人:Chester B. Whitley
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依托单位:
The Lysosomal Disease Network's 10th Annual WORLD Symposium
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批准号:8793924
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项目类别:
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资助金额:$2.0万
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财政年份:2013
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负责人:Chester B. Whitley
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依托单位:
The Lysosomal Disease Network's 10th Annual WORLD Symposium
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批准号:8648085
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项目类别:
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资助金额:$1.5万
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财政年份:2013
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network-8th Annual WORLD Symposium
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批准号:8312091
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项目类别:
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资助金额:$1.6万
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财政年份:2012
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network-9th Annual WORLD Symposium
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批准号:8456842
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项目类别:
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资助金额:$2.83万
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财政年份:2012
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负责人:Chester B. Whitley
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依托单位:
Administration
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批准号:8212824
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项目类别:
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资助金额:$10.88万
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财政年份:2011
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负责人:Chester B. Whitley
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依托单位:
WORLD Symposium 2010 (Lysosomal Disease Network's 6th Annual Research Meeting)
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批准号:7915961
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项目类别:
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资助金额:$3.5万
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财政年份:2010
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:8545226
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项目类别:
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资助金额:$124.87万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:8150442
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项目类别:
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资助金额:$124.42万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:9803937
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项目类别:
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资助金额:$123.92万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network Administrative Core
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批准号:10018658
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项目类别:
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资助金额:$26.41万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
RDCRC Administrative Unit
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批准号:7885746
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项目类别:
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资助金额:$11.21万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:7937808
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项目类别:
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资助金额:$139.23万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:10264848
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项目类别:
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资助金额:$112.76万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network Pilot Core
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批准号:10264854
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项目类别:
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资助金额:$7.27万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:8091863
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项目类别:
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资助金额:$10.73万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
WORLD Symposium 2009 (Lysosomal Disease Network's 5th Annual Research Meeting)
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批准号:7740100
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项目类别:
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资助金额:$2.0万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network
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批准号:10707869
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项目类别:
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资助金额:$112.51万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
Lysosomal Disease Network Administrative Core
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批准号:10707870
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项目类别:
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资助金额:$26.41万
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财政年份:2009
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负责人:Chester B. Whitley
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依托单位:
海外基金