Neurology
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We sought to examine prevalence and predictors of noninvasive ventilation (NIV) in a composite cohort of patients with amyotrophic lateral sclerosis (ALS) followed in a clinical trials setting (Pooled Resource Open-Access ALS Clinical Trials database). ⋯ NIV use was lower than expected in this ALS cohort that was likely to be optimally managed. Absence of respiratory symptoms and female sex may be barriers to NIV use. Prospective exploration of factors affecting adoption of NIV may help bridge this gap and improve care in ALS.
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Randomized Controlled Trial
Repetitive TMS does not improve cognition in patients with TBI: A randomized double-blind trial.
To determine whether high-frequency repetitive transcranial magnetic stimulation (rTMS) improves cognition in patients with severe traumatic brain injury. ⋯ This study provides Class II evidence that for individuals with chronic DAI, high-frequency rTMS over the left DLPFC does not significantly improve cognition.
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To determine whether preventive trials in genetic prion disease could be designed to follow presymptomatic mutation carriers to onset of disease. ⋯ The characterization of biomarkers suitable to serve as surrogate endpoints will be essential for the prevention of genetic prion disease. Parameters such as longer trial duration, increased enrollment, and the use of historical controls in a postmarketing study could provide opportunities for subsequent determination of clinical benefit.
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To find early hemodynamic predictors of outcome and reperfusion injury in patients with acute ischemic stroke due to anterior circulation large artery occlusion (LAO) after endovascular treatment (EVT). ⋯ Post-EVT ultrasound monitoring of stroke patients might be an effective bedside method for assessing treatment efficacy, shedding light on outcome variability and identifying patients at increased risk of ICH.
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Randomized Controlled Trial
Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis.
To investigate safety and explore efficacy of efgartigimod (ARGX-113), an anti-neonatal Fc receptor immunoglobulin G1 Fc fragment, in patients with generalized myasthenia gravis (gMG) with a history of anti-acetylcholine receptor (AChR) autoantibodies, who were on stable standard-of-care myasthenia gravis (MG) treatment. ⋯ This study provides Class I evidence that efgartigimod is safe and well-tolerated in patients with gMG.