Expert review of neurotherapeutics
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Expert Rev Neurother · Jan 2016
ReviewTau-directed approaches for the treatment of Alzheimer's disease: focus on leuco-methylthioninium.
Small molecular weight compounds able to inhibit formation of tau oligomers and fibrils have already been tested for Alzheimer's disease (AD) treatment. The most advanced tau aggregation inhibitor (TAI) is methylthioninium (MT), a drug existing in equilibrium between a reduced (leuco-methylthioninium) and oxidized form (MT(+)). MT chloride (also known as methylene blue) was investigated in a 24-week Phase II study in 321 mild-to-moderate AD patients at the doses of 69, 138, and 228 mg/day. ⋯ The dose of 138 mg/day showed potential benefits on cognitive performance of moderately affected patients and cerebral blood flow in mildly affected patients. A follow-up compound (TRx0237) claimed to be more bioavailable and less toxic than MT, is now being developed. Phase III clinical trials on this novel TAI in AD and in the behavioral variant of frontotemporal dementia are underway.
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Expert Rev Neurother · Jan 2016
ReviewCannabidiol as potential treatment in refractory pediatric epilepsy.
In recent years there has been great scientific and public interest focused on the therapeutic potential of compounds derived from cannabis for the treatment of refractory epilepsy in children. From in vitro and in vivo studies on animal models, cannabidiol (CBD) appears to be a promising anticonvulsant drug with a favorable side-effect profile. In humans, CBD efficacy and safety is not supported by well-designed trials and its use has been described by anecdotal reports. It will be necessary to investigate CBD safety, pharmacokinetics and interaction with other anti-epileptic drugs (AEDs) alongside performing double-blinded placebo-controlled trials in order to obtain conclusive data on its efficacy and safety in children.
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Expert Rev Neurother · May 2015
Meta AnalysisPrognostic value of copeptin in patients with acute stroke.
There is a need to improve stroke care through the prompt identification of stroke patients at increased risk of an adverse outcome. ⋯ Copeptin is an independent predictor of poor outcome and mortality for patients with acute stroke.
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Expert Rev Neurother · May 2015
ReviewMotor neuron disease-frontotemporal dementia: a clinical continuum.
Overlap between motor neuron disease (MND) and frontotemporal dementia (FTD) occurs at clinical, genetic and pathological levels, and has been recently strengthened through the discovery of the C9orf72 genetic expansion. MND is now considered to be a multisystem disorder in which cognitive involvement may be present and, in some cases, may evolve to frank FTD. Identifying cognitive features in MND can be challenging, while, similarly, motor dysfunction in FTD may be overlooked. As such, the present review aims to decipher the variety of overlapping clinical features across the MND-FTD continuum.
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Sarcoidosis is a multi-organ immune-mediated disease, which manifests as neurosarcoidosis (NS) in approximately 10% of all affected patients. The diagnosis of NS requires a high degree of suspicion as well as histological confirmation. Neurological symptoms in patients with systemic sarcoidosis should not be assumed to be due to NS unless proven true. ⋯ The goals of available treatments include: halting inflammation, prevention of disease worsening and restoring neurological functions whenever possible. With immunosuppression, clinical remission of NS occurs in the majority of patients. However, in some others, the disease may still progress, as no permanent cure is yet available.