Quantitative Motor (Q-Motor) Assessments Suggest a Beneficial Central Effect of Laquinimod in a Phase II Study in Huntington Disease (LEGATO-HD)
Objective: Evaluate the change from baseline at week 52 in Q-Motor (Quantitative Motor) measures, exploratory, standardized, and rater-independent outcomes in the LEGATO-HD study. Background: LEGATO-HD…Subcortical atrophy is an early event in Huntington’s disease
Objective: To determine whether brain regional volumes are associated with clinical symptoms in premanifest and manifest Huntington’s disease (HD). Background: Striatal atrophy has been considered a…NBIA-like MRI findings in a patient with Huntington’s disease
Objective: To report a rare case of Huntington’s disease (HD) with MRI findings resembling neurodegeneration with brain iron accumulation (NBIA). Background: HD and NBIA are…A safety, tolerability and biomarker update from an ongoing open-label extension study of RG6042 in adults with early manifest Huntington’s disease
Objective: To present the safety, tolerability and biomarker effects of RG6042 (previously, IONIS-HTTRX) during an open-label extension (OLE) study in adults with early manifest Huntington’s…Usefulness of Heart Rate Variability to Identify the Risk of Falling in Huntington’s Disease
Objective: To evaluate the relationship between the HRV and the risk of falling in HD patients. Background: Huntington's disease (HD) patients have a high prevalence…Digital monitoring of Huntington’s disease with smartphone and smartwatch wearable technology: The Digital-HD study
Objective: To investigate the tolerability and feasibility of using wearable smartphone and smartwatch technologies to monitor the motor, behavioural, cognitive and quality of life (QoL)…Changes in GABAergic transmission of striatal neurons in presymptomatic Huntington’s Disease
Objective: To determine whether parameters of synaptic and extrasynaptic GABAergic inhibition of striatal projection neurons are altered in presymptomatic BACHD mice. Background: One attractive early…Novel exploratory outcome assessments in GENERATION HD1
Objective: To improve collection of outcomes meaningful to patients with Huntington’s disease (HD) and their families, through the development of novel assessments of daily functional…Using functional status to aid interpretation of cUHDRS scores in patients with Huntington’s disease
Objective: To enhance understanding of the composite Unified Huntington’s Disease Rating Scale (cUHDRS) scoring by linking to established measures of meaningful daily function and independence…Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy
Objective: To assess the translatability and long-term efficacy of gene therapy-mediated huntingtin (HTT)-lowering in a large animal model of Huntington disease (HD), transgenic HD (tgHD)…
