Huntington’s disease in Egypt; A big challenge for a mysterious disease
Objective: To identify the challenges of managing Huntington’s disease in Egypt in order to overcome them. Background: Huntington’s disease (HD) is considered a rare disease…Healthcare resource utilisation and costs among patients with versus without Huntington’s disease in the US population
Objective: Compare the marginal healthcare resource utilisation (HRU) and costs between US patients with Huntington’s disease (HD) and non-HD controls. Background: HD is a genetic,…Deutetrabenazine for Huntington’s disease chorea—A Single Center’s Experience
Objective: To evaluate real-world use and outcomes with deutetrabenazine for Huntington’s disease. Background: Grade A evidence for Huntington’s disease chorea treatment includes tetrabenazine and deutetrabenazine.…Evaluation of the Safety of Deutetrabenazine at Higher Doses to Treat Chorea in Huntington’s Disease
Objective: To evaluate the safety and tolerability of deutetrabenazine >48 mg/d compared to ≤48 mg/d to treat Huntington’s disease (HD)–associated chorea in ARC-HD. Background: In…VMAT 2 inhibitor and Antipsychotic use in Individuals with Huntington’s Disease
Objective: To retrospectively investigate whether the concomitant use of VMAT2 inhibitors and antipsychotics in individuals with Huntington's disease (HD) is associated with an increased occurrence…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)…
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