Analysis of Participant Withdrawal in Huntington Disease Clinical Trials [0.03%]
亨廷顿舞蹈病临床试验中研究对象脱落的分析
Haruhiko Banno,Kelly L Andrzejewski,Michael P McDermott et al.
Haruhiko Banno et al.
Published Erratum
Journal of Huntington's disease. 2017;6(4):371. DOI:10.3233/JHD-179002 2017
Safety and Exploratory Efficacy at 36 Months in Open-HART, an Open-Label Extension Study of Pridopidine in Huntington's Disease [0.03%]
开放标签延展研究open-HART中36个月的安全性及探索性疗效分析：普瑞冬平治疗亨廷顿舞蹈病患者的疗效分析
Andrew McGarry,Karl Kieburtz,Victor Abler et al.
Andrew McGarry et al.
Background: Open-HART is an open-label extension of HART, a randomized, placebo-controlled, dose-ranging, parallel-group study. Objective: ...
Randomized Controlled Trial
Journal of Huntington's disease. 2017;6(3):189-199. DOI:10.3233/JHD-170241 2017
Sai S Chaganti,Elizabeth A McCusker,Clement T Loy
Sai S Chaganti
Background: Although the typical age of onset for Huntington's disease (HD) is in the fourth decade, between 4.4-11.5% of individuals with HD have a late onset (over 60 years of age). Diagnosis of Late onset HD (LoHD) can...
Haruhiko Banno,Kelly L Andrzejewski,Michael P McDermott et al.
Haruhiko Banno et al.
Background: Excellent retention in Huntington disease (HD) clinical trials is essential for testing new therapies. The stage of disease, cognitive status, and availability of a care partner may influence retention in HD c...
Fifteen Years of Clinical Trials in Huntington's Disease: A Very Low Clinical Drug Development Success Rate [0.03%]
亨廷顿舞蹈病临床试验15年：非常低的临床药物研发成功率
André M Travessa,Filipe B Rodrigues,Tiago A Mestre et al.
André M Travessa et al.
Background: Drug development in Huntington's disease (HD) is particularly challenging, and only two compounds are approved by the FDA. It is therefore essential to appraise drug development programs in order to understand...
Dynamic Prediction of Motor Diagnosis in Huntington's Disease Using a Joint Modeling Approach [0.03%]
亨廷顿病的联合建模方法动态预测运动诊断变化
Kan Li,Erin Furr-Stimming,Jane S Paulsen et al.
Kan Li et al.
Background: Prediction of motor diagnosis in Huntington's disease (HD) can be improved by incorporating other phenotypic and biological clinical measures in addition to cytosine-adenine-guanine (CAG) repeat length and age...
Amyloid Precursor Protein Haploinsufficiency Preferentially Mediates Brain Iron Accumulation in Mice Transgenic for The Huntington's Disease Mutation [0.03%]
载脂蛋白前体蛋白单倍型功能不全选择性介导亨廷顿舞蹈病转基因小鼠脑铁蓄积
Kiersten Berggren,Sonal Agrawal,Julia A Fox et al.
Kiersten Berggren et al.
Background: Huntington's disease (HD) is an autosomal dominant disorder caused by a CAG expansion in the huntingtin gene that results in expression of mutant huntingtin protein. Iron accumulates in HD brain neurons. Amylo...
Assessing Decline: Visualising Progression in Huntington's Disease using a Clinical Dashboard with Enroll-HD Data [0.03%]
基于Enroll-HD数据的临床仪表盘在亨廷顿舞蹈病进展评估中的应用研究
Thomas Walker,Boyd Ghosh,Christopher Kipps
Thomas Walker
Background: In Huntington's disease (HD), it remains unclear how symptom severity and rate of symptomatic change relates to age and CAG repeat number (CAGn). It is often difficult for clinicians to assess whether an affec...
Female Sexual Dysfunction in Presymptomatic Mutation Carriers and Patients with Huntington's Disease [0.03%]
亨廷顿舞蹈病前驱期及患者女性性功能障碍的特点
Matej Kolenc,Jan Kobal,Simon Podnar
Matej Kolenc
Background: Although in Huntington's disease (HD) movement, cognition, and personality are most significantly affected, autonomic dysfunction should not be neglected. In women with HD sexual dysfunction has not been adequ...