First-in-Human Trial Begins for an Antisense Oligonucleotide in KCNT1-Related Epilepsy Marco Meglio

Published Date: 21 Oct 2026

A phase 1b/2 trial is testing an intrathecal antisense oligonucleotide designed to lower KCNT1 expression in children with a severe, drug-resistant epilepsy syndrome.

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Latest News

Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.

Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 16, 2026.

Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.

Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nicholas J. Maragakis, MD; and Jinsy Andrews, MD, MSc, FAAN, FANA. [LISTEN TIME: 23 minutes]

News All Time

Telitacicept Shows Deepening, Durable Response in Myasthenia Gravis Marco Meglio

New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.

Individualizing Advanced Therapies in Parkinson Disease: Hubert Fernandez, MD Hubert Fernandez, MD, Louie Pasculli

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NeurologyLive® Brain Games: October 11, 2026 Louie Pasculli

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Carmela Tartaglia, MD, FRCPC, discusses the challenges of diagnosing frontotemporal dementia, the evolving role of biomarkers, and emerging approaches to disease modification.

NeurologyLive® Friday 5 — October 9, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 9, 2026.

Phase 4 THRIVE Data Highlights Eptinezumab’s Impact on Migraine After Prior CGRP Failure Marco Meglio

Newly announced Phase 4 THRIVE trial results showed most patients who previously had an inadequate response to a CGRP-targeting therapy improved with eptinezumab over 24 weeks.

FDA Action Update, September 2026: Approvals, Clearances, and Priority Review Isabella Ciccone, MPH

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Solengepras Meets Primary Endpoint in Phase 3 ARISE Trial of Parkinson Disease Marco Meglio

Phase 3 ARISE trial results show solengepras 150 mg significantly reduced OFF time and improved motor and non-motor symptoms in Parkinson disease.

CSF Biomarkers Identify Shared LRRK2 Pathway Activation in Sporadic and LRRK2-Associated Parkinson Disease Louie Pasculli

New cerebrospinal fluid biomarker data suggest shared LRRK2 pathway activation across sporadic and LRRK2-associated Parkinson disease, potentially informing biomarker development and therapeutic targeting.

Metabolic Agent Vutiglabridin Improves Motor Scores in Early-Stage Study of Parkinson Disease Marco Meglio

Phase 2a trial results showed 800 mg vutiglabridin significantly improved motor symptom scores compared with placebo in early-stage Parkinson's disease.

Christen Kutz, PhD, PA-C, on What Real-World Data Reveal About Treatment Transitions to Nipocalimab in Generalized Myasthenia Gravis Marco Meglio, Christen Kutz, PhD, PA-C

Christen Kutz, PhD, PA-C, discusses a retrospective survey of neurology providers examining why patients with generalized myasthenia gravis are transitioning to nipocalimab and what real-world outcomes data suggest about its role in the treatment landscape.

New Study Reveals Plasma Biomarkers Highlight Distinct Biological Responses to Lecanemab in Alzheimer Disease Louie Pasculli, Carlos Cruchaga, PhD

Carlos Cruchaga, PhD, director of the NeuroGenomics and Informatics Center at Washington University, discusses emerging blood-based biomarkers for monitoring biological and cognitive responses to lecanemab treatment in Alzheimer disease.

Brain Chat: The Overlap Years: Perimenopause, Menopause, and MS Mitzi Joi Williams, MD

Mitzi Joi Williams, MD, is joined by Jakai McEwen, DO, and Charnetta Colton-Poole, MD, to discuss the challenges of communication between patients and their health care teams, including navigating medical language, promoting shared decision-making, and advocating for clear, patient-centered care.

Advanced Therapies in Parkinson Disease: Matching the Right Treatment to the Right Patient Hubert Fernandez, MD, Louie Pasculli

An MDS 2026 plenary speaker discusses the evolving role of infusion therapies, deep brain stimulation, and focused ultrasound in Parkinson disease, including when to consider device-aided therapies and how treatment selection can be individualized.

Emerging Advances in Parkinson Disease Ahead of MDS 2026: Benjamin Walter, MD, MBA Benjamin L. Walter, MD, Louie Pasculli

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