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

Published Date: 17 Oct 2026

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.

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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.

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.

ELEVIDYS Shows Sustained Functional Differences vs External Controls in Older Ambulatory Duchenne Patients Louie Pasculli

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

Dompé Begins Phase 2 Trial Testing Intranasal Nerve Growth Factor for Cerebral Palsy Marco Meglio

The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

Todays Updates

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]

Todays Updates

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]

Latest News

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.

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.

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

News All Time

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.

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.

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.

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.

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.

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.

Clinical Forum Highlights Shifting Paradigm in NF1 Plexiform Neurofibroma Care Marco Meglio

At a NeurologyLive Clinical Forum in Philadelphia, Jessica D. Schulte, MD, PhD, of NYU Grossman School of Medicine, discussed how earlier recognition and systemic therapy options are changing the treatment paradigm for patients with NF1-associated plexiform neurofibromas.

Ecopipam Shows Early and Sustained Tic Reduction in New Pediatric Tourette Syndrome Analyses Louie Pasculli

New analyses from the D1AMOND clinical program found that 69.8% of participants achieved a clinically meaningful reduction in tic severity within 8 weeks of ecopipam treatment, while an interim 18-month analysis showed sustained improvement without new safety signals.

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