April 17th 2025
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.
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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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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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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.
Revolutionizing Neurology Care: Previewing Cleveland Clinic’s New Neurological Institute
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.
Challenges in Treating Genetically Predisposed Alzheimer Disease: Insights from APOLLOE4
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.
Next-Gen Gene Editing for Muscular Dystrophy: Exploring the ARCUS Approach
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.
Phase 3 Study to Test 1st Generation Antihistamine EPX-100 in Lennox-Gastaut Syndrome
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.
Phase 3 INFRONT-3 Study Details Baseline Characteristics of Participants With FTD-GRN
April 8th 2025A 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.