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
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/7594728
• 关键词: Androgen Antagonists; Antiandrogen Therapy; Biological Markers; Clinical Research; Clinical Trials; Development; Disease; Dose; Dutasteride; Friedreich Ataxia; Goals; Inherited; Patients; Phase II Clinical Trials; Purpose; Research; Safety; Series; Spinal Muscular Atrophy; Testing; idebenone; mouse model; nervous system disorder; programs; spinal and bulbar muscular atrophy

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).

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。