April 24th 2025
Roche has updated its phase 3 GENERATION HD2 trial to continue testing only the higher 100 mg dose of tominersen, following interim data favoring its potential clinical benefit in Huntington disease.
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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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Final CHAMPION MG Trial Data Show Sustained Benefit, Safety of Ravulizumab in Myasthenia Gravis
April 22nd 2025Results from the open-label extension of a phase 3 trial showed that treatment with ravulizumab sustained clinical benefits for up to 4 years in patients with generalized myasthenia gravis.
Breaking Down Oculopharyngeal Muscular Dystrophy: From Clinical Gaps to Genetic Gains
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.
Real-World Data Show Similar Comorbidity Rates With and Without Sodium Oxybate Treatment
April 21st 2025Data 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.
How Personalized Repetitive Transcranial Magnetic Stimulation May Transform Alzheimer Treatment
Ken Mariash, chief executive officer at Sinaptica, provided commentary on new data from the company’s SinaptiStim neuromodulation system, highlighting its clinical promise in slowing Alzheimer disease progression through targeted, non-drug brain network stimulation.
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.