Journal of neurology, neurosurgery, and psychiatry
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J. Neurol. Neurosurg. Psychiatr. · Dec 2015
Observational StudyShort-interval observational data to inform clinical trial design in Huntington's disease.
To evaluate candidate outcomes for disease-modifying trials in Huntington's disease (HD) over 6-month, 9-month and 15-month intervals, across multiple domains. To present guidelines on rapid efficacy readouts for disease-modifying trials. ⋯ To exploit the potential power of specific neuroimaging measures such as caudate atrophy in disease-modifying trials, we propose their use as (1) initial short-term readouts in early phase/proof-of-concept studies over 6 or 9 months, and (2) secondary end points in efficacy studies over longer periods such as 15 months.
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J. Neurol. Neurosurg. Psychiatr. · Dec 2015
Validation of biomarkers in subcortical ischaemic vascular disease of the Binswanger type: approach to targeted treatment trials.
Vascular cognitive impairment (VCI) is a heterogeneous group of cerebrovascular diseases secondary to large and small vessel disease. We hypothesised that biomarkers obtained early in the disease could identify a homogeneous subpopulation with small vessel disease. ⋯ Biomarkers predicted the diagnosis of the BD type of subcortical ischaemic vascular disease. Using pathophysiological biomarkers to select homogeneous groups of patients needs to be tested in targeted treatment trials.
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J. Neurol. Neurosurg. Psychiatr. · Dec 2015
Antifibroblast growth factor receptor 3 antibodies identify a subgroup of patients with sensory neuropathy.
Immunological mechanisms are suspected in sensory neuropathy (SN) occurring with systemic autoimmune diseases and in some idiopathic cases, but so far there are no antibodies (Abs) identifying these neuropathies. ⋯ A anti-FGFR3 Abs identify a subgroup of patients with SN in whom an underlying autoimmune disorder affecting sensory neurons in the dorsal root and trigeminal nerve ganglia is suspected.
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J. Neurol. Neurosurg. Psychiatr. · Dec 2015
The temporal evolution of structural and functional measures after acute optic neuritis.
In this prospective case series, we aimed to characterise the temporal evolution of functional and structural measures in the afferent visual pathway of patients with acute optic neuritis (ON). ⋯ Structural and functional measures evolve over time in patients with ON. There may be sex-specific differences in recovery after an acute ON event.