
The phase 1 LUMINA trial evaluates Amylyx Pharmaceuticals' AMX0114, an antisense oligonucleotide targeting calpain-2, as a potential therapy for ALS by focusing on safety, tolerability, and biomarkers tied to disease progression.
The phase 1 LUMINA trial evaluates Amylyx Pharmaceuticals' AMX0114, an antisense oligonucleotide targeting calpain-2, as a potential therapy for ALS by focusing on safety, tolerability, and biomarkers tied to disease progression.
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.
Neurology News Network. for the week ending April 12, 2025. [WATCH TIME: 3 minutes]
Phase 2b trial results showed Descartes-08, a CAR-T therapy, achieved sustained efficacy and a strong safety profile in myasthenia gravis, with notable responses in biologic-naïve patients and no major safety concerns over 12 months.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 11, 2025.
Experts discussed recent advancements in diagnostic criteria, imaging techniques, and the evolving understanding of multiple sclerosis subtypes and treatment responses. [WATCH TIME: 10 minutes]
Cleveland Clinic’s $1.1 billion Neurological Institute, opening in 2027, will revolutionize neurological care with a state-of-the-art, patient-centered design integrating advanced technology and innovative care solutions.
In one of the longest trials of brain stimulation in Alzheimer disease, repetitive transcranial magnetic stimulation led to a reduced decline in CDR-SB, as well as positive impacts on cognitive function and functional abilities.
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 prefilled syringe is approved for a 20- to 30-second subcutaneous injection, and patients can self-inject following proper instruction in the subcutaneous injection technique.
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]
Catch up on any of the neurology news headlines you may have missed over the course of March 2025, compiled all into one place by the NeurologyLive® team.
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.
The professor of neurology at the University of Texas Health Science Center San Antonio discussed some of the challenges with therapeutic development for OPMD, as well as advances and barriers to gene therapy approaches. [WATCH TIME: 4 minutes]
The clinical associate professor of neurology and neurosurgery at NYU Langone Health emphasized the importance of rapid, targeted blood pressure reduction and bundled care to improve outcomes in patients with intracerebral hemorrhage. [WATCH TIME: 4 minutes]
Relative to previously approved therapies like rimegepant, ubrogepant, and zavegepant, AXS-07 resulted in better 2-hour pain relief, sustained pain relief, and reduced use of rescue medications.
The Melissa and Paul Anderson President’s Distinguished Professor of Neurology, Perelman School of Medicine, University of Pennsylvania discussed evolving insights into the cellular immunology of multiple sclerosis. [WATCH TIME: 4 minutes]
Harmony Biosciences’ phase 3 study will assess EPX-100, a repurposed antihistamine, as a potential treatment for Lennox-Gastaut syndrome, targeting seizure reduction and improved outcomes.
The clinical professor of stroke neurology at Stanford University discussed the clinical rationale and evidence supporting early, intensive blood pressure lowering in patients with intracerebral hemorrhage.
A phase 3 trial investigating latozinemab, a monoclonal antibody therapy for frontotemporal dementia because of GRN mutations, reported baseline participant characteristics to better characterize this patient population.
Murali Doraiswamy, MD, MBBS, a professor of psychiatry and geriatrics at Duke University School of Medicine, gave clinical follow-up on a pivotal analysis of the phase 3 APOLLOE4 study testing a novel therapeutic for Alzheimer disease.
Newly presented open-label extension results at AAN 2025 suggest that investigational ZYN002, a cannabidiol gel, may help improve irritability in children and adolescents living with Fragile X syndrome.
The staff scientist at Gladstone Institutes talked about leveraging AI-driven behavioral analysis to better model and assess Alzheimer disease progression in preclinical studies. [WATCH TIME: 2 minutes]
Findings from a phase 3 study revealed that patients with Parkinson disease switching to IPX203 had greater benefits in good ON time when also taking lower doses of dopamine agonists.
The professor of neuroscience at the Imperial College London discussed the emerging role of GLP-1 analogs as neuroprotective agents with potential benefits in neurodegenerative conditions. [WATCH TIME: 4 minutes]
Denali Therapeutics has begun submitting a BLA for tividenofusp alfa as a potential Hunter syndrome treatment, supported by promising biomarker reductions in clinical trials.
The director of the UCLA Goldberg Migraine Program discussed the advantages, evidence base, and evolving payor support for CGRP-targeting therapies as potentially a first-line option in migraine prevention. [WATCH TIME: 5 minutes]
The clinical professor of stroke neurology and neurocritical care at Stanford University advocated for early and intensive blood pressure reduction in patients with intracerebral hemorrhage to limit hematoma expansion and improve outcomes. [WATCH TIME: 5 minutes]
Teva’s migraine therapy fremanezumab (Ajovy) may soon expand its reach to pediatric patients, as the FDA reviews a supplemental application for its use in children and adolescents aged 6 to 17.
The ENABLE phase 4 study will evaluate the real-world safety, efficacy, and patient experience of ublituximab in relapsing multiple sclerosis over a 96-week period.