Seeking regulatory feedback on novel clinical outcome assessments: a case example from the Huntington’s Disease – Regulatory Science Consortium (HD-RSC)
Objective: To align with regulators on the utility and development strategy of novel clinical outcome assessments for use in Huntington’s disease (HD) clinical trials. Background:…Motor assessments in HD clinical trials: Q-Motor versus the UHDRS-TMS – what did we learn from recent studies?
Objective: To review the performance of UHDRS-TMS and Q-Motor quantitative motor measures in recent placebo-controlled clinical trials. Background: The UHDRS-TMS has widely been used to…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,…Huntington’s Disease presenting as sporadic cerebellar ataxia
Objective: To describe a unique case of a woman who presented as a progressive cerebellar ataxia, with no family history of neurological diseases, that after…A global survey on speech language therapists’ awareness to Huntington’s disease
Objective: To investigate the awareness and knowledge of speech and language therapists (SLTs) on Huntington’s disease (HD) worldwide, and to explore the clinical practice and…Permanent chorea in 76-years-old female with poorly controlled diabetes
Objective: To present a unique case of a patient with hyperglycemic associated chorea showing no improvement during a long-term follow-up, despite a strict glycemic control.…Multimodal characterization of the visual network in Huntington’s disease
Objective: To examine the relationship between structural organization, functional processing and task performance of the visual system in a cohort of Huntington’s disease (HD) gene-mutation…A life-threatening case of Anti-IgLON5 disease
Objective: To present a case of anti-IgLON5 disease with life-threatening respiratory complications. Background: Anti-IgLON5 disease is a little known CNS disorder characterized by non-REM and…Can we hear pathology in premanifest Huntington’s disease? Acoustic and listener-based analysis of speech
Objective: The present study evaluated the ability of acoustic and listener-based perceptual measures to differentiate speech deficits in the premanifest and early stages of HD…Burden of Huntington’s disease in the US: Analysis of an insured population
Objective: To describe the healthcare resource utilization (HRU) in Huntington’s disease (HD) in private (commercial) and public (Medicare and Medicaid) insurance plans in the US.…
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