The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]
Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.
The neuroradiologist at Mayo Clinic discussed the clinical and conceptual differences between Alzheimer disease diagnostic frameworks and how clinicians should prepare for a shifting treatment landscape. [WATCH TIME: 4 minutes]
Sonya Miller, medical director at TauRx, discussed the latest clinical data and development plans for HMTM, a potential oral therapy targeting tau pathology in Alzheimer disease.
Neuromuscular expert Matthew Alexander, PhD, explores the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.
The resident in the Department of Neurology at NYU Langone Health discussed the preliminary findings of a 9-month study on lecanemab recently presented at the 2025 AAN Annual Meeting. [WATCH TIME: 2 minutes]
The former FDA commissioner discussed the collaborative power of the neuromuscular community and the challenges of implementing emerging therapies in a rapidly evolving care landscape. [WATCH TIME: 4 minutes]
The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.
The executive vice president of research at the National MS Society talked about how the award continues to honor transformative multiple sclerosis research and inspire the next generation of investigators. [WATCH TIME: 5 minutes]
The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations. [WATCH TIME: 4 minutes]
Philip Kremer, MD, Research Director Neurology, Center for Human Drug Research, provided clinical insights on the early-stage progress of ARV-102, an LRRK2 degrader, in LRRK2-associated diseases.
A duo of neurology experts from Feinberg School of Medicine talked about the diagnostic challenges, evolving understanding, and clinical implications of paraneoplastic neurological syndromes. [WATCH TIME: 3 minutes]
The professor in the Department of Pathology and Laboratory Medicine at the University of California, Irvine, gave clinical insights on efforts to refine neuropsychological outcome measures for individuals with Down syndrome at risk for Alzheimer disease. [WATCH TIME: 3 minutes]
The chief scientific officer at Satellos Bioscience discussed phase 1 findings on SAT-3247, an oral AAK1 inhibitor, highlighting its safety, pharmacokinetics, and early translatability in Duchenne muscular dystrophy. [WATCH TIME: 7 minutes]
The director of NYU Langone's Multiple Sclerosis Comprehensive Care Center discussed the importance of early, high-efficacy treatment for pediatric MS. [WATCH TIME: 3 minutes]
In a late-stage trial, telitacicept demonstrated significant and rapid clinical improvements in gMG, achieving notable symptom reductions, improved quality of life, and strong safety outcomes.
The chief medical officer at Cognito Therapeutics provided a clinical overview on promising findings surrounding the company’s gamma sensory stimulation device in patients with Alzheimer disease. [WATCH TIME: 10 minutes]
High doses of RNS60 led to greater number of patients with modified Rankin scale scores between 0-2, improved EQ-5D-5L index score, and enhanced NIHSS score at each pre-specified time point over placebo.
The vice president of research at Cognition Therapeutics provided commentary on the safety of investigational CT1812 and its role in immune response and synapse-related pathways. [WATCH TIME: 3 minutes]
Elizabeth Head, PhD, a professor in the Department of Pathology and Laboratory Medicine at the University of California, Irvine, shed light on the emerging research and trials surrounding the interplay between Alzheimer disease and Down syndrome.
In late-breaking findings from the TRANQUIL study presented at AAN 2025, the wearable demonstrated significant improvements in tremor reduction and daily functioning over a sham device.
The senior director of mission programs at The ALS Association discussed how the thinkALS Toolkit can aid general neurologists in identifying and referring suspected cases more efficiently. [WATCH TIME: 5 minutes]
Patients treated with efgartigimod for myasthenia gravis were found to have a higher incidence and severity of infections compared to those treated with immunoglobulins.
The physician assistant at UCSF Movement Disorder and Neuromodulation Center reflected on the value of AMDAPP in fostering professional connection, education, and patient-centered care among movement disorder APPs. [WATCH TIME: 3 minutes]
ARV-102 showed dose-dependent LRRK2 degradation and favorable safety in its first-in-human trial, supporting its promise for treating LRRK2-linked neurodegenerative diseases.
Experts discussed recent advancements in diagnostic criteria, imaging techniques, and the evolving understanding of multiple sclerosis subtypes and treatment responses. [WATCH TIME: 10 minutes]
Emer MacSweeney, MD, a consultant neuroradiologist and trial investigator of the phase 3 APOLLOE4 study, provided a clinical view of the latest study findings, and the difficulties with finding treatments for APOEε4/4 carriers of Alzheimer disease.
The professor of neurology at the University of Pennsylvania Perelman School of Medicine discussed the current research landscape, clinical challenges, and future directions for noninvasive neuromodulation techniques in neurorehabilitation. [WATCH TIME: 4 minutes]
The neuroradiologist at Mayo Clinic provided clinical insights on the controversy with two recently published Alzheimer criteria, with one diagnosing the disease based on biomarkers alone vs clinical symptoms. [WATCH TIME: 4 minutes]
Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, its innovative approach to treating Duchenne muscular dystrophy, and promising preclinical data presented at MDA 2025.
