Tumor Pathology in Patients with Huntington’s Disease in a Specialized Center of Buenos Aires, Argentina (Preliminary Data)
Objective: To analyze the prevalence and type of neoplasms in patients with HD and to contrast it with the data of the general population in…Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington’s Disease
Objective: To extrapolate the human equivalent dose (HED) for a Phase I/II clinical trial in adults to explore the safety, tolerability, and efficacy of striatally-administered…MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates
Objective: To assess the effects of intra-striatal administration of a huntingtin protein (HTT) lowering gene therapy in macaca fascicularis non-human primates (NHP). Background: The aggregation…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…
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