课题基金 / 基金详情

CLINICAL TRIAL: EXTENSION STUDY - LATE ONSET POMPE DISEASE

CLINICAL TRIAL: EXTENSION STUDY - LATE ONSET POMPE DISEASE
临床试验：扩展研究 - 迟发性庞贝病
批准号：
7724970
负责人：
BRUCE A BARSHOP
金额：
$0.82万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN DIEGO
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
项目状态：
已结题
起止时间：
2007-12-01 至 2008-11-30
来源：
https://reporter.nih.gov/project-details/7724970
关键词：
Clinical TrialsComputer Retrieval of Information on Scientific Projects DatabaseDouble-Blind MethodFundingGlycogen storage disease type IIGrantInstitutionOrphan DiseasePatientsPlacebo ControlResearchResearch PersonnelResourcesSafetySourceUnited States National Institutes of Health

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中文摘要
翻译
这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 评估Myozyme治疗迟发性庞培病患者的长期安全性和有效性，这些患者以前在安慰剂对照的双盲研究AGLU02704下接受治疗。孤儿疾病状况。
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. Assess the long term safety and efficacy of Myozyme treatment in patients with late onset Pompe disease who were previously treated under the placebo-controlled, double blind study AGLU02704. Orphan disease status.
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