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Treatment of pediatric drug-resistant generalized epilepsy with responsive neurostimulation of the centromedian nucleus of the thalamus: A case series of seven patients.
Epilepsy Res
January 2025
Division of Pediatric Neurology, Department of Pediatrics, University of Pittsburgh School of Medicine, Children's Hospital of Pittsburgh, 8th Floor Faculty Pavilion, 4401 Penn Ave., Pittsburgh, PA 15224, United States. Electronic address:
Purpose: Responsive neurostimulation of the centromedian nucleus of the thalamus (CM RNS) is being investigated for treatment of drug-resistant generalized epilepsy with promising results. The aim of this study is to report outcomes of seven patients with pediatric-onset drug-resistant generalized epilepsy, including both genetic generalized epilepsy (GGE) and Lennox-Gastaut syndrome (LGS), who underwent treatment with bilateral CM RNS.
Methods: A retrospective chart review was performed for patients with drug-resistant generalized epilepsy who underwent treatment with bilateral CM RNS at Children's Hospital of Pittsburgh from 2020 to 2022.
Development of a Patient Reported Outcome Measure of Side Effects for Patients Taking Calcineurin Inhibitors: The FACIT-CNI-Ntx.
Am J Kidney Dis
January 2025
Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, USA; Northwestern University Transplant Outcomes Research Collaborative, Comprehensive Transplant Center, Feinberg School of Medicine, Chicago, IL, USA; Centre for Patient Reported Outcomes Research, Department of Applied Health Sciences, University of Birmingham, Edgbaston, Birmingham, UK.
Rationale & Objective: Valid measures of side effects are important to inform clinical use of calcineurin inhibitors (CNIs). This study sought to develop and establish the content validity of a PRO measure to capture side effects among kidney transplant recipients taking CNIs.
Study Design: Qualitative interviews for concept elicitation and cognitive debriefing.
Intranasal oxytocin for apathy in people with frontotemporal dementia (FOXY): a multicentre, randomised, double-blind, placebo-controlled, adaptive, crossover, phase 2a/2b superiority trial.
Lancet Neurol
February 2025
Department of Clinical Neurological Sciences, University of Western Ontario, London, ON, Canada; Department of Cognitive Neurology, St Joseph's Health Care London, London, ON, Canada. Electronic address:
Background: No treatments exist for apathy in people with frontotemporal dementia. Previously, in a randomised double-blind, placebo-controlled, dose-finding study, intranasal oxytocin administration in people with frontotemporal dementia improved apathy ratings on the Neuropsychiatric Inventory over 1 week and, in a randomised, double-blind, placebo-controlled, crossover study, a single dose of 72 IU oxytocin increased blood-oxygen-level-dependent signal in limbic brain regions. We aimed to determine whether longer treatment with oxytocin improves apathy in people with frontotemporal dementia.
View Article and Find Full Text PDFSafety and efficacy of satralizumab in patients with generalised myasthenia gravis (LUMINESCE): a randomised, double-blind, multicentre, placebo-controlled phase 3 trial.
Lancet Neurol
February 2025
Department of Neurology, International University of Health and Welfare, Narita, Japan.
Background: Evidence from preclinical studies suggests that IL-6 signalling has the potential to modulate immunopathogenic mechanisms upstream of autoantibody effector mechanisms in patients with generalised myasthenia gravis. We aimed to assess the safety and efficacy of satralizumab, a humanised monoclonal antibody targeting the IL-6 receptor, in patients with generalised myasthenia gravis.
Methods: LUMINESCE was a randomised, double-blind, placebo-controlled, multicentre, phase 3 study at 105 sites, including hospitals and clinics, globally.
Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study.
Lancet Neurol
February 2025
Janssen Research & Development, a Johnson & Johnson Company, Titusville, NJ, USA.
Background: Given burdensome side-effects and long latency for efficacy with conventional agents, there is a continued need for generalised myasthenia gravis treatments that are safe and provide consistently sustained, long-term disease control. Nipocalimab, a neonatal Fc receptor blocker, was associated with dose-dependent reductions in total IgG and anti-acetylcholine receptor (AChR) antibodies and clinically meaningful improvements in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale in patients with generalised myasthenia gravis in a phase 2 study. We aimed to assess the safety and efficacy of nipocalimab in a phase 3 study.
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