January 13th 2025
Recent research investigated how HSV-1 infection affects tau phosphorylation through the cGAS-STING-TBK1 pathway, highlighting potential new therapeutic strategies for Alzheimer disease.
Get direct access to interviews with experts, as well as the latest data and clinical news updates, straight from the conference floor in Los Angeles.
Individualizing Treatment for Patients with Generalized Myasthenia Gravis
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A New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient Burden
March 1, 2025
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Patient, Provider & Caregiver Connection™: Reducing the Burden of Parkinson Disease Psychosis with Personalized Management Plans
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Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
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Burst Expert Illustrations & Commentary™: Visualizing the Role of FcRN in Neurological Disorders
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BURST Expert Illustrations and Commentaries™: Visualizing FcRn as a Therapeutic Target in Neurological Disease
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BURST Expert Illustrations and Commentaries™: Visualizing the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis
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Clinical Consultations™: Navigating the Evolving Treatment Landscape in Generalized Myasthenia Gravis
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Epilepsy Agent RAP-219 Shows Promising Safety, Target Occupancy Across Phase 1 Studies
An 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.
Rising Clinical Utility of Plasma P-Tau217 in Diagnosing Alzheimer Disease
Masoud 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
Posdinemab, 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 Accepts Amended Protocol for Phase 3 Trial of Buntanetap in Alzheimer Disease
The revised protocol merges the 6-month symptomatic and 18-month disease-modifying studies into a single trial with a 6-month symptomatic readout and a 12-month disease-modifying evaluation.
FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia
SGT-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
The 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.
DNL343 Falls Short of Primary End Point in Phase 2/3 HEALEY-ALS Platform Trial
Over a 24-week period, DNL343 failed to distinguish itself from placebo on the primary end point of ALSFRS-R, as well as on other secondary end points of muscle strength and respiratory function.
Reducing the Burden: Proactive Care Strategies for Lennox-Gastaut Syndrome
Babitha 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.
Phase 3b SMART Study Findings Complement Real-World Evidence for Zolgensma in SMA
Most participants showed improvements in motor function, with 61% achieving a clinically meaningful increase in HFMSE scores by week 52 and 41% achieving similar gains in RULM scores.
Axsome to Submit NDA for AXS-05 in Alzheimer Agitation Following Positive Phase 3 Trials
The ACCORD-2 trial demonstrated a significant reduction in overall worsening compared to placebo, while the ADVANCE-2 trial showed numerically greater improvements without reaching statistical significance.
FDA Removes Partial Clinical Hold on VG-3927 for Microglial Dysfunction in Alzheimer Disease
January 6th 2025Results from Vigil Neuroscience’s ongoing phase 1 trial of VG-3927 in healthy volunteers supported the continued development of the therapy as a potential once-daily oral treatment for Alzheimer disease.