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Studies Of Hereditary Neurological Disease: Clinical Trials

Studies Of Hereditary Neurological Disease: Clinical Trials
遗传性神经系统疾病的研究：临床试验
批准号：
7594728
负责人：
Kenneth Fischbeck
金额：
$135.32万
依托单位：
NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
依托单位国家：
美国
项目类别：
财政年份：
--
资助国家：
美国
项目状态：
未结题
起止时间：
至
来源：
https://reporter.nih.gov/project-details/7594728
关键词：
Androgen AntagonistsAntiandrogen TherapyBiological MarkersClinical ResearchClinical TrialsDevelopmentDiseaseDoseDutasterideFriedreich AtaxiaGoalsInheritedPatientsPhase II Clinical TrialsPurposeResearchSafetySeriesSpinal Muscular AtrophyTestingidebenonemouse modelnervous system disorderprogramsspinal and bulbar muscular atrophy

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中文摘要
翻译
该研究项目的目的是为遗传性神经系统疾病开发安全有效的治疗方法。过去一年的具体研究成果包括：（1）建立脊髓性肌萎缩可靠生物标志物的合作研究，（2）完成高剂量艾地苯醌治疗弗里德赖希共济失调的2期研究，以及（3）开发2期临床试验，以测试度他雄胺治疗脊髓和延髓肌萎缩（肯尼迪病）的可行性。
英文摘要
The purpose of this research program is to develop safe and effective treatments for hereditary neurological disorders. Specific research accomplishments in the past year include the following: (1) a collaborative study to establish reliable biomarkers for spinal muscular atrophy, (2) completion of a phase 2 study of high dose idebenone treatment for Friedreich's ataxia, and (3) development of a phase 2 clinical trial to test the feasibility of dutasteride treatment for spinal and bulbar muscular atrophy (Kennedy's disease).
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Studies of Hereditary Neurological Disease: Disease Mechanisms
Studies Of Hereditary Neurological Disease: Disease Gene Identification
Studies Of Hereditary Neurological Disease: Clinical Trials
Studies Of Hereditary Neurological Disease: Disease Gene Identification
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