The Roche HD Natural History Study – an external comparator by design
Objective: The Roche Natural History (NH) study in Huntington’s disease (HD) (NCT03664804) is a prospective, longitudinal cohort study designed to provide further clinical validation of…Exploring the effects of intrastriatal AAV5-miHTT therapy on MRS signal and mutant huntingtin levels in the Q175FDN mouse model of HD
Objective: To assess the effects of intrastriatal AAV5-miHTT therapy on MRS signal and mutant huntingtin levels in the Q175FDN mouse model of HD. Background: Huntingtin…Huntington’s disease: A case review of Sub-Saharan Africans with diverse origins
Objective: To characterise the Huntingtons disease phenotype in Sub-saharan Africans. Background: Huntington’s disease (HD) is a debilitating neurodegenerative illness that leads to diminished cognitive function,…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)…Ancillary Service Utilization and Impact in Huntington’s Disease
Objective: To determine which Huntington’s disease (HD) patient characteristics correlate with ancillary service utilization. To examine the longitudinal impact of ancillary therapies on clinical characteristics.…Pallidal surgery and physiological mechanism in choreic movements
Objective: To investigate the effect of deep brain stimulation (DBS) or coagulation of the internal globus pallidus (GPi) for choreic movements of various disorders and…Steroid responsive chorea in Moyamoya disease: illustrative cases
Objective: To report the rapid reduction of disabling chorea in two cases of pediatric Moyamoya disease with pulse steroid therapy. Background: The common clinical presentation…Internal pallidum stimulation reduces mobile generalized dystonia in Wilson’s disease
Objective: Globus Pallidus internus (GPi) deep brain stimulation (DBS) is established in treatment of dystonia. We evaluated safety and efficacy of bilateral GPi stimulation in…Feasibility and acceptability of a therapist-delivered physical activity coaching intervention for people with premanifest and early stage Huntington’s Disease and Parkinson’s Disease
Objective: To assess the feasibility, acceptability, and preliminary efficacy of the Pre-Active PD and HD interventions for increased physical activity (PA) engagement in people with…Electroconvulsive Therapy (ECT): An Effective Option for the Treatment of Medication-Resistant Psychiatric Symptoms in Huntington’s Disease: A Case Series and Review of the Literature
Objective: Through a single site case series, we investigate the use and effectiveness of electroconvulsive therapy (ECT) for the treatment of refractory psychiatric symptoms in…
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