Epilepsy Agent RAP-219 Shows Promising Safety, Target Occupancy Across Phase 1 Studies
January 12th 2025An ongoing phase 2a study of RAP-219 involves adult patients with drug-resistant epilepsy who have an implanted responsive neurostimulation (RNS) device, allowing for real-time monitoring of intracranial EEG data.
Discovering a New Receptor for Nerve Growth Factor: Nigel Bunnett, PhD, BSc
January 10th 2025The professor and chair of the Department of Molecular Pathobiology at NYU Langone provided a clinical overview of preclinical research identifying a new receptor for nerve growth factor that plays an important role in pain signaling. [WATCH TIME: 4 minutes]
Rising Clinical Utility of Plasma P-Tau217 in Diagnosing Alzheimer Disease
January 9th 2025Masoud Toloue, chief executive officer at Quanterix, and Nicholas J. Ashton, PhD, senior director of the Banner Health Fluid Biomarker Program, discussed the Simoa technology required to test for p-tau217, the expanding understanding of this biomarker, and the next steps for advancing this research.
FDA Grants Fast Track Designation to Anti-Tau Therapy Posdinemab
January 9th 2025Posdinemab, a monoclonal antibody that targets the mid-domain of Alzheimer disease-specific phosphorylated tau, is currently being investigated in a placebo-controlled phase 2b trial that employs a plasma biomarker as a screening tool.
FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia
January 8th 2025SGT-212 delivers full-length frataxin via MRI-guided intradentate nucleus infusion and intravenous administration to target mitochondrial dysfunction in neurons and cardiomyocytes, addressing both neurologic and cardiac symptoms.
Stoke Announces Phase 3 EMPEROR Registrational Study for Zorevunersen in Dravet Syndrome
January 8th 2025The global Phase 3 trial aims to enroll ~150 patients with Dravet syndrome who have SCN1A variants, assessing zorevunersen's impact on seizure frequency, behavior, cognition, and safety over 60 weeks, with results expected by 2027.
Overlapping Diagnoses and the Advancement of MS Detection in NMDARE: Joseph Kuchling, MD
January 7th 2025The postdoctoral research assistant at Charité University Berlin discussed advances in diagnostic criteria and 7-Tesla MRI imaging, highlighting a previously overlooked overlap between anti-N-methyl-D-aspartate receptor encephalitis and multiple sclerosis. [WATCH TIME: 5 minutes]
Reducing the Burden: Proactive Care Strategies for Lennox-Gastaut Syndrome
January 7th 2025Babitha Haridas, MD, MBBS, a pediatric epileptologist at Johns Hopkins Medicine, discussed the complexities with managing status epilepticus in Lennox-Gastaut syndrome, focusing on the importance of identifying triggers early in their condition.
Advancing Future Drug Development for Narcolepsy: Mihaela Nowak, MD
January 6th 2025The neurologist and sleep disorder specialist at Allegheny Health Network provided an in-depth exploration of the current advancements and future directions in narcolepsy drug development. [WATCH TIME: 3 minutes]