AAN 2025 – Editor’s picks
Explore the key abstracts we’re most excited to hear about at the 77th American Academy of Neurology (AAN) Annual Meeting, which is taking place in San Diego, CA, and online, from April 5-9, 2025.
Epilepsy
Safety, Tolerability, and Efficacy of Bexicaserin in a Cohort of Participants With Developmental and Epileptic Encephalopathies: Interim Results of a Phase 1b/2a PACIFIC Study Open-Label Extension
Author: Ngoc Minh Le, et al.
First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy – Updated Clinical Trial Results
Author: John Hixson, et al.
A Modern Design for a Phase 2/3 Randomized, Double-blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of BHV-7000 in Idiopathic Generalized Epilepsy with Generalized Tonic-Clonic Seizures
Author: Jason Lerner, et al.
Responsive Neurostimulation and Focal Cortical Dysplasia: Personalizing Technology to Pathology in Drug-Resistant Epilepsy
Author: Vincent Chang, et al.
Headache
Efficacy and Safety of Fremanezumab for the Preventive Treatment of Episodic Migraine in Children and Adolescents: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study
Author: Andrew D. Hershey, et al.
Efficacy and Safety of a First-in-Class Investigational Prescription Digital Therapeutic for Episodic Migraine (CT-132): Phase 3 Double-Blind, Randomized, Controlled Trial
Author: Cassandra Snipes, et al.
BHV-2100, a First-in-Class TRPM3 Antagonist in Development for the Treatment of Migraine
Author: Beth Emerson, et al.
Real-World Long-Term Effectiveness of Fremanezumab in Patients With Migraine Who Switched From Another mAb Targeting the CGRP Pathway – Subgroup Analysis of the FINESSE Full Analysis
Author: Andreas Straube, et al.
Comparative Efficacy of AXS-07 vs. Gepants for Acute Treatment of Migraine: A Network Meta-Analysis
Author: Stephanie J Nahas, et al.
Movement disorders
Parkinson’s
Systemic AAV Gene Therapy Using a CNS-targeted Engineered Capsid Significantly Increases GCase Activity to Support the Potential Treatment of PD-GBA
Author: Reed Ressler, et al.
Solengepras (CVN424): A Novel GPR6 Inhibitor in Clinical Development as an Investigational Therapy for Parkinson’s Disease
Author: Kelvin Chou, et al.
Parkinson’s Disease Mild Cognitive Impairment with MRI evidence of Cholinergic Nucleus 4 Degeneration: A New Subtype?
Author: Ahmed Negida, et al.
Efficacy and Safety of Tavapadon, an Orally Administered, Once-daily, Selective D1/D5 Partial Dopamine Agonist, Adjunctive to Levodopa for Treatment of Parkinson’s Disease with Motor Fluctuations
Author: Hubert Fernandez, et al.
Safety, Tolerability, and Clinical Assessment of Bemdaneprocel for Parkinson’s Disease: Results Through 24 Months From a Phase 1 Study
Author: Harini Sarva, et al.
Multiple System Atrophy & Essential Tremor
Topline Data from a Randomized, Double Blind, Placebo Controlled Phase 2 Study of ATH434 in Multiple System Atrophy
Author: David Stamler, et al.
Cerebellar Repetitive Transcranial Magnetic Stimulation for Essential Tremor: A Systematic Review and Meta-analysis
Author: David Abraham, et al.
Update on the Long-term Safety of Magnetic Resonance-guided Focused Ultrasound in Essential Tremor: Comprehensive Review of Outcomes from Clinical Trials and Real-world Experience to Date
Author: Amit Sokolov, et al.
Huntington’s & CSF1R-related Disorders
Sustained Chorea Improvements with Long-term, Once-daily Valbenazine in Adults with Huntington’s Disease
Author: Erin Furr Stimming, et al.
The Phase 3 PROOF-HD Trial Demonstrates Meaningful Benefits of Pridopidine on Function, Cognition, and Motor Signs in Huntington Disease (HD)
Author: Michal Geva, et al.
Using 7-Tesla MRI as a Biomarker for CSF1R-related Disorders: Preliminary Results
Author: Tomasz Chmiela, et al.
Evaluating the Utility of Biomarker in Assessing Symptoms of Patients with CSF1R-related Disorders
Author: Tomasz Chmiela, et al.
Multiple Sclerosis
A Phase 1, Open-label, Single Center Study of KYV-101, an Autologous Fully-human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Non-relapsing and Progressive Forms of Multiple Sclerosis
Author: Jeffrey Dunn, et al.
Predictive Value of the Combination of CSF and Serum Biomarkers at Disease Onset in Multiple Sclerosis
Author: Enric Monreal Laguillo, et al.
Tolebrutinib Versus Teriflunomide in Relapsing Multiple Sclerosis: Efficacy and Safety Results from the Phase 3 GEMINI 1 and 2 Trials
Author: Jiwon Oh, et al.
Evaluation of CHIT1 and SERPINA3 as Candidate Cerebrospinal Fluid Biomarkers of Progressive Biology in Multiple Sclerosis
Author: Amit Bar-Or, et al.
Real-world Effectiveness and Predictors of Response to Autologous Haematopoietic Stem Cell Transplantation in MS: The UK Experience in 271 Patients Treated During 2000-2023
Author: Gavin Brittain, et al.
Detecting Smouldering Multiple Sclerosis with Real-world Mobility Monitoring
Author: Gavin Brittain, et al.
Neuromuscular Disorders
Myasthenia Gravis
Solutions to Address the Unmet Needs of the gMG Patient Journey in the US: A Multistakeholder Delphi Consensus Study
Author: Judith Thompson, et al.
Design of KYSA-6, a Phase 2, Open-label, Multicenter Study of KYV-101, a Novel Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy in Refractory Generalized Myasthenia Gravis
Author: Aiden Haghikia, et al.
Efficacy of Nipocalimab, a Novel Neonatal Fragment Crystallizable Receptor Blocker, as Measured Using Quantitative Myasthenia Gravis Assessment: Findings from the Phase 3 Placebo-controlled Vivacity-MG3 Study
Author: Sindhu Ramchandren, et al.
Efficacy and Safety of Autologous BCMA-directed mRNA CAR T-Cell Therapy in Generalized Myasthenia Gravis: Results from a Phase 2b Randomized Placebo-controlled Trial
Author: Tuan Vu, et al.
Phase 3 Myasthenia Gravis Inebilizumab Trial (MINT): Efficacy and Safety Results in Patients with Generalized MG
Author: Richard Nowak, et al.
Amyotrophic Lateral Sclerosis
RAG-17, a Novel siRNA Therapy for SOD1-ALS: Safety and Preliminary Efficacy from a First-in-human Trial
Author: Jinyi Ye, et al.
Diagnostic Value of Cerebral Peduncle Diameter in Amyotrophic Lateral Sclerosis Using Transcranial Ultrasound
Author: Armin Ariaei, et al.
Post-onset ALSFRS-R Slope (POS) as a Predictor of Disease Progression in Patients with Amyotrophic Lateral Sclerosis (ALS)
Author: Larisa Ibric, et al.
Spinal Muscular Atrophy
CSF and Blood Neuronal Injury Biomarkers in Spinal Bulbar Muscular Atrophy and Amyotrophic Lateral Sclerosis 4
Author: Pashtun-Poh Shahim, et al.
Improving Bone Health Screening in Patients with Spinal Muscular Atrophy: A Quality Improvement Initiative
Author: Erin McCoy, et al.
Safety and Efficacy of Disease-modifying Therapies in Spinal Muscular Atrophy: A Systematic Review and Network Meta-analysis
Author: Mohamed Mamdouh, et al.
Duchenne Muscular Dystrophy & Other NMDs
Safety and Efficacy of DYNE-101 in Adults with DM1: Phase 1/2 ACHIEVE Trial Data
Author: Daniel Wolf, et al.
Efficacy and Safety of Targeted Immunotherapy with ANX005 in Treating Guillain-Barré Syndrome: A Phase 3 Multicenter Study
Author: Henk-Andre Kroon, et al.
Pharmacokinetics, Pharmacodynamics (PK/PD), and Safety Profile of Ulviprubart: Results from a 48-week, Open-label, Phase 1 Study in Patients with Inclusion Body Myositis (IBM)
Author: Merrilee Needham, et al.
Neuropathies
Transition from Intravenous Immunoglobulin to Efgartigimod PH20 SC in Participants with Chronic Inflammatory Demyelinating Polyneuropathy: A Phase 4 Study in Progress
Author: Yessar Hussain, et al.
RNAi-based Therapies for Hereditary Transthyretin Amyloidosis with Polyneuropathy: A Meta-analysis of Randomized Clinical Trials
Author: Oguz Kagan Sahin, et al.
Sleep Disorders
Efficacy and Safety of TAK-861, an Oral Orexin Receptor 2 Agonist, in Individuals with Narcolepsy Type 1: Results from a Phase 2 Trial and Long-Term Extension Study Interim Analysis
Author: Yves Dauvilliers, et al.
Samelisant (SUVN-G3031): Positive Results from Phase-2 Proof-of-concept, Double-blind, Placebo-controlled Study in Patients with Narcolepsy and an Update on Further Clinical Development
Author: Ramakrishna Nirogi, et al.
AXS-12 for the Treatment of Narcolepsy: Topline Results from the Phase 3 SYMPHONY Trial
Author: Michael Thorpy, et al.
The Orexin 2 Receptor Agonist ALKS 2680 in Patients with Narcolepsy Type 1: An Initial Proof of Concept Phase 1b Study
Author: Ron Grunstein, et al.
Phase 1 Clinical Data with Orexin Receptor 2 (OX2R) Agonist, ORX750, in Acutely Sleep-Deprived Healthy Participants
Author: Deborah Hartman, et al.
Stroke
The Ultimate Stroke Scale (USS): An Innovative Tool for Automated Large Vessel Occlusion Screening
Author: Sachin Kothari, et al.
RESCUE: A Proof-of-Concept Trial with Adjunct RNS60 Treatment Shows Safety, Reduced Infarct Growth, and Numerical Improvement in all Prespecified Efficacy Endpoints in Participants with Acute Ischemic Stroke
Author: Supurna Ghosh, et al.
Tirofiban with Mechanical Thrombectomy in Endovascular Treatment of Stroke: A Meta-analysis
Author: Advaith Rao, et al.
Targeted versus High-Intensity Monitoring Following Intravenous Thrombolysis in Acute Ischemic Stroke
Author: Carl-Lewis Valcinord, et al.
Intravenous Thrombolysis Alone versus Endovascular Thrombectomy Alone versus Intravenous Thrombolysis plus Endovascular Thrombectomy for Ischemic Stroke Beyond the 4.5-Hour Window: A Network Meta-Analysis of Randomized Clinical Trials
Author: Marina Vilardo, et al.
Efficacy of Rescue Therapy After Failed Mechanical Thrombectomy in Acute Ischemic Stroke: A Systematic Review and Meta-Analysis
Author: Hany Atwan, et al.
