Levacetylleucine Shows Sustained Benefit at 3 Years in Niemann-Pick Disease Marco Meglio

Published Date: 19 Oct 2026

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.

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Episode 177: Previewing the 2026 NEALS Annual Meeting Marco Meglio, Jinsy A. Andrews, MD, MSc, Nicholas Maragakis, MD

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]

FDA Grants Fast Track Status to EG-501 in Neuropsychiatric Lupus Marco Meglio

The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

TSHA-102 Produces Multidomain Functional Gains in Rett Syndrome Matt Hoffman

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

Satralizumab Reduces MOGAD Relapse Risk in Phase 3 METEOROID Trial Louie Pasculli

Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.

New Phase 1 Study Tests Intrathecal Gene Therapy Agent INS1201 in Duchenne Muscular Dystrophy Marco Meglio

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

MDS Congress 2026: Late-Breaking Data Snapshot Marco Meglio

A quick-read roundup of 10 late-breaking abstracts from the 2026 MDS Congress, from phase 3 Parkinson disease readouts to long-term data in Huntington disease and Tourette syndrome, with the key results that matter for practice.

Todays Updates

Verekitug's Quarterly Dosing Cuts Asthma Burden, With Michael Wechsler, MD Michael Wechsler, MD, MSc

Wechsler discusses why less frequent dosing matters to patients with severe asthma, plus VALIANT's safety profile and what's next for the field.

Tozorakimab Cuts COPD Mucus Plugging, With George Washko, MD

Tozorakimab Cuts COPD Mucus Plugging, With George Washko, MD

Chronic Proton Pump Inhibitor Use Linked to More Asthma Exacerbations

Chronic Proton Pump Inhibitor Use Linked to More Asthma Exacerbations

FDA Approves Autoinjector for Self-Administered Zilucoplan in Generalized Myasthenia Gravis Marco Meglio

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

Recognizing Familial Chylomicronemia Syndrome in the APOC3 Era

Recognizing Familial Chylomicronemia Syndrome in the APOC3 Era

Todays Updates

Verekitug's Quarterly Dosing Cuts Asthma Burden, With Michael Wechsler, MD Michael Wechsler, MD, MSc

Wechsler discusses why less frequent dosing matters to patients with severe asthma, plus VALIANT's safety profile and what's next for the field.

Tozorakimab Cuts COPD Mucus Plugging, With George Washko, MD

Tozorakimab Cuts COPD Mucus Plugging, With George Washko, MD

Chronic Proton Pump Inhibitor Use Linked to More Asthma Exacerbations

Chronic Proton Pump Inhibitor Use Linked to More Asthma Exacerbations

FDA Approves Autoinjector for Self-Administered Zilucoplan in Generalized Myasthenia Gravis Marco Meglio

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

Recognizing Familial Chylomicronemia Syndrome in the APOC3 Era

Recognizing Familial Chylomicronemia Syndrome in the APOC3 Era

Latest News

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]

The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

A quick-read roundup of 10 late-breaking abstracts from the 2026 MDS Congress, from phase 3 Parkinson disease readouts to long-term data in Huntington disease and Tourette syndrome, with the key results that matter for practice.

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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

Catch up on any of the neurology headlines you may have missed in September 2026, compiled into 1 place by the NeurologyLive® team.

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.

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.

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.

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.

GLP-1/GIP Agonist KP405 Shows Biomarker Signal in Parkinson Disease Marco Meglio

Exploratory extracellular vesicle biomarkers suggested KP405, a brain-penetrant GLP-1/GIP agonist, engaged insulin signaling and neuroinflammation pathways in a small phase 1 study.

12-Month Gluten-Free Diet Linked to Cognitive, Quality-of-Life Improvements in Parkinson Disease Louie Pasculli

Late-breaking findings from MDS 2026 suggest a gluten-free diet may affect cognitive and quality-of-life measures in Parkinson disease, although no significant changes in motor function were observed.

From Cord Blood to Cannabinoids: 7 Agents to Watch in Cerebral Palsy Marco Meglio

Seven investigational cerebral palsy therapies—from baby-tooth stem cells to cannabinoids and botulinum toxin—show mixed early results, highlighting key clinical trial milestones ahead.

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

Benjamin Walter, MD, MBA, discusses how advances in biomarker testing and genetic characterization are informing Parkinson disease care, alongside developments in adaptive deep brain stimulation and efforts to identify disease earlier.

Oral D1/D2 Agonist Lu AF28996 Increases Good ON-Time in Phase 1b Advanced PD Study, Phase 2 DARE2 Trial Begins Isabella Ciccone, MPH

Investigational oral dopamine agonist prodrug boosts good ON-time, cuts OFF-time and dyskinesia, and reduces levodopa in advanced Parkinson disease, as phase 2 begins.

NeuroVoices: Kamalini Galvelis, MS, on Why Diverse Cohorts Matter in Replicating a GBA1 Intronic Risk Variant Isabella Ciccone, MPH, Kamalini Galvelis, MS

The associate vice president of clinical research at the Parkinson's Foundation discussed a large-scale replication of rs3115534-G, a noncoding GBA1 variant tied to Parkinson disease in people of African ancestry.

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