A longitudinal PET study to assess the state of microglia activation in a Phase 2 study of Laquinimod as a treatment for Huntington’s disease (LEGATO-HD)
Objective: To assess in vivo in Huntington’s disease (HD) changes in microglia activation state, due to treatment with laquinimod, as reflected by changes in 11C-PBR28…How Well Does the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) Reflect Disease Progression in Huntington’s disease (HD)?
Objective: To examine the usefulness of the cUHDRS in reflecting disease progression in a well-characterized group of mild to moderate HD subjects followed at one…Evaluation of Cortical Excitability in Huntington’s Disease and Parkinson’s Disease
Objective: To evaluate the cortical excitability difference in Huntington's disease (HD) and Parkinson's disease (PD) using paired-pulse transcranial magnetic stimulation (TMS). Background: TMS is useful…Translatable biomarkers in gene therapy for Huntington’s Disease: learnings from pre-clinical studies
Objective: We have developed AAV5-miHTT, a recombinant AAV-based gene therapy expressing an engineered microRNA that specifically binds to HTT exon1, resulting in lowering of both…Speech is a sensitive marker in premanifest Huntington’s disease: exploring acoustic, cognitive and fine motor performance
Objective: To explore the utility of objective analysis of speech as a quantifiable clinical biomarker in premanifest HD. Background: The autosomal dominant inheritance of Huntington’s…Remote and frequent assessment of Huntington’s disease in clinical trials: Strategies for assessing and accounting for the practice effect
Objective: To assess the impact of task repetition on performance in digital assessments on Huntington’s disease (HD) and establish the number of practice test iterations…The roles of Huntingtin Associated Protein 40 in Huntingtin functions and Huntington’s disease pathogenesis
Objective: Huntington’s disease (HD) is caused by an abnormal expansion of a glutamine tract (polyQ) in huntingtin (HTT), a large scaffold protein with numerous reported…Biological and Clinical Manifestations of Huntington’s disease in Gene Carriers Very Far from Predicted Onset: The Young Adult Study
Objective: To perform a deep phenotyping of a Huntington’s Disease (HD) cohort further from onset than previously studied to establish when disease related markers first…Defining time and anatomical specificity of basal ganglia white matter loss in premanifest Huntington’s disease
Objective: To identify when white matter connections first begin to degenerate in HD and which connections are most susceptible to early degeneration. Background: Huntington’s disease…A Novel Case of Persistent Segmental Spinal Myoclonus Due to Spinal Astrocytoma Temporarily Relieved by Diversion of Cerebrovascular Fluid
Objective: A 36-years-old woman with a spinal cord diffuse intramedullary astrocytoma WHO grade II, with multifocal brain parenchymal involvement. After insertion of her ventriculo-peritoneal shunt…
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