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CLINICAL TRIAL: A PHASE II STUDY OF PN401 PYRIMIDINE RESPONSIVE SYNDROME OF NEUR

CLINICAL TRIAL: A PHASE II STUDY OF PN401 PYRIMIDINE RESPONSIVE SYNDROME OF NEUR
临床试验:PN401 嘧啶反应性神经综合征综合征的 II 期研究
批准号:
7950917
负责人:
BRUCE A BARSHOP
金额:
$0.11万
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
翻译
该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者(PI)可能从另一个NIH来源获得主要资金, 因此可以在其他CRISP条目中表示。列出的机构是 中心,不一定是研究者的机构。 评价PN 401在神经功能障碍和感染易感性的嘧啶反应综合征患者中减少癫痫发作和感染、改善言语和语言以及认知功能的安全性和疗效,评价生活质量,并描述PN 401对综合征体征和症状的影响。
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. Evaluate safety and efficacy of PN401 in reduction of seizures and infections, improvement of speech and language and cognitive functions in patients with pyrimidine responsive syndrome of neurological dysfunction and susceptibility to infection, evaluate quality of life, and describe effects of PN401 on signs and symptoms of the syndrome.
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会议论文
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CLINICAL TRIAL: A PHASE II STUDY OF PN401 PYRIMIDINE RESPONSIVE SYNDROME OF NEUR
CLINICAL TRIAL: ALGLUCOSIDASE ALFA TEMPORARY ACCESS PROGRAM (ATAP) PROTOCOL AGLU
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