Lancet neurology
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Randomized Controlled Trial Multicenter Study
Efficacy and safety of rifampicin for multiple system atrophy: a randomised, double-blind, placebo-controlled trial.
No available treatments slow or halt progression of multiple system atrophy, which is a rare, progressive, fatal neurological disorder. In a mouse model of multiple system atrophy, rifampicin inhibited formation of α-synuclein fibrils, the neuropathological hallmark of the disease. We aimed to assess the safety and efficacy of rifampicin in patients with multiple system atrophy. ⋯ National Institutes of Health, Mayo Clinic Center for Translational Science Activities, and Mayo Funds.
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Deep-brain stimulation (DBS) of the subthalamic nucleus (STN) is an established treatment for motor complications in Parkinson's disease. 20 years of experience with this procedure have contributed to improved understanding of the role of the STN in motor, cognitive, and emotional control. In Parkinson's disease, the pathological STN neuronal activity leads to motor, cognitive, and emotional inhibition. ⋯ Conversely, the notable reduction in anti-parkinsonian drug dose allowed by motor improvement can unveil mesolimbic hypodopaminergic behaviours such as apathy, anxiety, or depression. Fine-tuning of stimulation parameters with dopaminergic drugs is necessary to prevent or improve pathological behaviours.
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Sex chromosome aneuploidies are a common group of disorders that are characterised by an abnormal number of X or Y chromosomes. However, many individuals with these disorders are not diagnosed, despite established groups of core features that include aberrant brain development and function. ⋯ Continuing attention to emerging trends in research of sex chromosome aneuploidies is important for clinicians because it informs appropriate management of these common genetic disorders. Furthermore, improved understanding of underlying neurobiological processes has much potential to elucidate sex-related factors associated with neurological and psychiatric disease in general.
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Randomized Controlled Trial Multicenter Study
Effect of oral cladribine on time to conversion to clinically definite multiple sclerosis in patients with a first demyelinating event (ORACLE MS): a phase 3 randomised trial.
Patients who develop relapsing-remitting multiple sclerosis (MS) present with a first clinical demyelinating event. In this double-blind, multicentre, randomised, phase 3 study we investigated the effect of oral cladribine on conversion to clinically definite MS in patients with a first clinical demyelinating event, when given at the same doses shown to be effective in relapsing-remitting MS. ⋯ Merck Serono SA Geneva, a subsidiary of Merck KGaA, Darmstadt, Germany.