Mena Farag,Sarah J Tabrizi,Edward J Wild
Mena Farag
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Ther...
Upregulation of endocannabinoid signaling in vivo restores striatal synaptic plasticity and motor performance in Huntington's disease mice [0.03%]
体内上调内源大麻素信号转导可恢复亨廷顿舞蹈病小鼠纹状体突触可塑性和运动表现能力
Marja D Sepers,Cameron L Woodard,Daniel Ramandi et al.
Marja D Sepers et al.
BackgroundSynaptic dysfunction underlies early sensorimotor and cognitive deficits in Huntington's disease (HD) and precedes the degeneration of striatal spiny projection neurons and cortical pyramidal neurons. Movement selection and motor ...
Functional Rating Scale 2.0 (FuRST 2.0): A patient-reported outcome measure of function for Huntington's disease [0.03%]
亨廷顿病功能测量量表2.0（ FuRST 2.0）：用于评价亨廷顿病患者报告的功能转归的工具
Rebecca Lm Fuller,Pua Feigenbaum,Nancy LaPelle et al.
Rebecca Lm Fuller et al.
BackgroundCurrent functional rating scales are not sensitive to the earliest functional changes in Huntington's disease (HD).ObjectiveThe Functional Rating Scale 2.0 (FuRST 2.0) is a patient-reported outcome (PRO) measure designed to be sen...
Asymmetric brain atrophy in Huntington's disease: A postmortem MRI study [0.03%]
亨廷顿舞蹈病的不对称脑萎缩：一项尸检MRI研究
Eardi Lila,David Hunt,Daniel D Child et al.
Eardi Lila et al.
BackgroundHuntington's disease is a progressive, autosomal dominant, neurodegenerative disease caused by a CAG repeat expansion in the HTT gene. Medium spiny neurons of the striatum are especially vulnerable to the disease, and atrophy of t...
A systematic review of performance-based functional capacity measures for use in Huntington's disease and evaluation of their suitability for clinical trials [0.03%]
基于性能的功能容量测量在亨廷顿病中的系统评价及其临床试验适用性的评估
Tayela M Prichard,Cali M Roiboit,Meg E Rankin et al.
Tayela M Prichard et al.
BackgroundHuntington's disease (HD) leads to a decline in functional capacity, affecting daily life tasks. Assessing functional capacity in clinical trials is crucial to evaluate treatment effectiveness and substantiate the clinical meaning...
Emerging roles of microglia and neuroinflammation in Huntington's disease: From pathophysiology to clinical trials [0.03%]
小胶质细胞和神经炎症在Huntington病中的新角色：从病理生理学到临床试验
Muna Abedrabbo,Pardis Kazemian,Colúm Connolly et al.
Muna Abedrabbo et al.
Microglia, the resident immune cells of the central nervous system, play a pivotal role in the response to Huntington's disease (HD) pathology. Through both cell-autonomous mechanisms and exposure to external pathogenic stimuli, microglia t...
Wenzhen Duan,Yuan Zhou,Hongshuai Liu
Wenzhen Duan
The glymphatic system, a macroscopic waste clearance network in the brain, plays a vital role in maintaining neuronal health and brain homeostasis. Functionally analogous to the lymphatic system in other organs, the term "glymphatic" combin...
Deutetrabenazine treatment outcomes with doses above U.S. Food and Drug Administration maximum approved doses in Huntington's disease chorea: A dual-site analysis [0.03%]
Huntingtons病舞蹈症状的氘化四苯甲酰胺治疗效果：一个双点位分析 在美国食品药品监督管理局最大批准剂量以上剂量的治疗效果分析
Kayla Dodson,Sabrina Livezey,Brittany Denson et al.
Kayla Dodson et al.
BackgroundDeutetrabenazine, a vesicular monoamine transporter 2 inhibitor, is one of few treatment options available for Huntington's disease (HD) chorea. There is limited data describing clinical experience with deutetrabenazine doses >48 ...
Comparative analysis of neurofilament light chain in Huntington's disease like 2 and Huntington's disease [0.03%]
亨廷顿舞蹈病样2型和亨廷顿舞蹈病的神经丝轻链比较分析
David G Anderson,Aline Ferreira-Correia,Filipe B Rodrigues et al.
David G Anderson et al.
Huntington's disease-like 2 (HDL2) closely resembles Huntington's disease (HD) in clinical and pathological features. Neurofilament light chain (NfL) is an important biomarker in HD research and holds potential in HDL2. To evaluate NfL's ut...