April 21st 2025
Data suggest no significant differences in comorbidity rates in a newly published study, highlighting dosing challenges and treatment patterns in patients receiving immediate-release sodium oxybate.
Get direct access to interviews with experts, as well as the latest data and clinical news updates, straight from the conference floor in San Diego.
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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Burst Expert Illustrations & Commentary™: Visualizing the Role of Subcutaneous Infusion as an Alternate Administration Route for Medical Interventions
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SimulatED™: Diagnosing and Treating Alzheimer’s Disease in the Modern Era
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SimulatED™: Understanding the Role of Genetic Testing in Patient Selection for Anti-Amyloid Therapy
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Expert Illustrations & Commentaries™: New Targets for Treatment in Cognitive Impairment in Schizophrenia – The Role of NMDA Receptors and Co-agonists
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From Fibrosis to Function: Exploring New Pathways in Muscular Dystrophy Research
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.
Michael Flanagan, PhD, on Del-zota’s Breakthrough Potential for Duchenne Muscular Dystrophy
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.
miRNA-214 Shows Potential as Biomarker to Predict ALS Disease Progression
Using discovery and validation cohorts, plasma miRNA-214 levels were shown to correlate with disease severity and were also associated with plasma neurofilament light and CSF inflammatory cytokines.
A Clinician’s Perspective on First-in-Human Data for LRRK2 Degrader ARV-102
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.
Large Danish Cohort Study Reveals No Significant Association Between HLA Alleles and Migraine
April 16th 2025Although the study reported no evidence linking human leukocyte antigen alleles to migraine, the findings were not replicated, suggesting the HLA system may not be involved in migraine susceptibility.
Afterimage Duration Significantly Longer in Migraine With Aura Than Without
Afterimage duration was more prolonged in patients with migraine with aura than those without, suggesting differing underlying pathophysiology between the two subtypes despite the exact mechanisms remaining unclear.
FDA Approves CT-132 as First Digital Therapeutic for Preventive Treatment of Episodic Migraine
CT-132, an approach that aims to reduce brain hypersensitivity, becomes the first marketed digital treatment to prevent migraine, intended to be used with other previously approved medications.
Investigational Telitacicept Significantly Improves Myasthenia Gravis Outcomes in Phase 3 Study
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.
Phase 2 RESCUE Data Show RNS60 Reduces Stroke Infarct and Improves Outcomes
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.
A New Era in Research: Exploring Cognitive Health and Alzheimer Disease in Down Syndrome
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.
Amylyx Begins Phase 1 LUMINA Study of CAPN2-Targeting Agent for ALS
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.
CAR-T Therapy Descartes-08 Demonstrates Efficacy, Safety in Phase 2 Trial of Myasthenia Gravis
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.