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

Published Date: 08 Oct 2026

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.

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Phase 3 PROPEL Trial to Test Imeroprubart in Generalized Myasthenia Gravis Marco Meglio

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Todays Updates

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

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

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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

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Latest News

Full results from the phase 2b LUMA showed that investigational BIIB122/DNL151 did not slow confirmed clinical worsening in early-stage Parkinson's disease compared with placebo.

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.

Here's some of what is coming soon to NeurologyLive® this week.

PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.

Early phase 1 findings showed that the PROTAC LRRK2 degrader ARV-102 was associated with improvements in saccadic hypometria and changes in CSF markers of endolysosomal function, neuroinflammation, synaptic integrity, and axonal guidance in participants with Parkinson disease.

A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.

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LGMD Agent BBP-418 Improves Ambulation and Pulmonary Function Across Subgroups in Phase 3 FORTIFY Interim Analysis Isabella Ciccone, MPH

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

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PETUNIA Study to Track Nipocalimab Use in Pregnancy Marco Meglio

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Vanda to Pursue sNDA for Tasimelteon in Delayed Sleep-Wake Phase Disorder After Positive Phase 3 Readout Isabella Ciccone, MPH

In a pivotal phase 3 study, once-daily tasimelteon 20 mg shifted sleep onset 37.1 minutes earlier than placebo in adults with delayed sleep-wake phase disorder.

Single Infusion of Rese-Cel Yields Meaningful Responses Off Immunomodulators in Phase 1/2 RESET-MG Trial Isabella Ciccone, MPH

Findings from the phase 1/2 RESET-MG trial, presented at AANEM 2026, showed that 10 of 13 patients with refractory generalized myasthenia gravis had clinically meaningful MG-ADL improvements after a single infusion of rese-cel.

Evaluating Zeleciment Basivarsen in Myotonic Dystrophy Type 1: The Phase 3 HARMONIA Trial Isabella Ciccone, MPH

The phase 3 HARMONIA trial will evaluate whether a muscle- and CNS-targeted antisense conjugate improves function across multiple systems in patients with myotonic dystrophy type 1.

Zilucoplan Autoinjector Matches Bioequivalence of Pre-Filled Syringe in Myasthenia Gravis Marco Meglio

New data from the 2026 AANEM Annual Meeting showed zilucoplan delivered via autoinjector was bioequivalent to the pre-filled syringe and was well tolerated when self-administered by patients with generalized myasthenia gravis.

Global Phase 3 UPSTREAM MG Trial of Telitacicept Advances Enrollment in Generalized Myasthenia Gravis Isabella Ciccone, MPH

The ongoing phase 3 study is enrolling approximately 180 adults across more than 13 countries, with topline results expected in mid-2027.

New Phase 3 Trials to Test Empasiprubart Against Placebo, IVIG in CIDP Louie Pasculli

Phase 3 trials EMNERGIZE and EMVIGORATE will evaluate the efficacy and safety of intravenous empasiprubart in adults with chronic inflammatory demyelinating polyradiculoneuropathy.

EpilepsyGTx, Viralgen Partner on AAV Gene Therapy EPY201 Ahead of Planned First-in-Human Trial Louie Pasculli

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NeuroVoices: James Winkley, MD, on Analyzing Phase 3 PREVAIL Trial of Gefurulimab Marco Meglio

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Somnolence Risk, Short-Term Efficacy Drive VMAT2 Inhibitor Choice in Older Adults With Tardive Dyskinesia Isabella Ciccone, MPH

In a discrete choice experiment, somnolence risk and short-term efficacy were the top factors driving vesicular monoamine transporter 2 inhibitor selection for tardive dyskinesia in patients aged 55 years and older.

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FDA Approves Tavapadon for the Treatment of Parkinson Disease Isabella Ciccone, MPH

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