Awareness, Not Access, Remains the Barrier to NF1 Therapy Marco Meglio

Published Date: 14 Nov 2026

At a NeurologyLive Clinical Forum in Phoenix, Kevin J. Bielamowicz, MD, of Arkansas Children’s Hospital, discussed how a lack of adult neuro-oncology infrastructure is shaping how NF1-associated plexiform neurofibromas get recognized, referred, and treated.

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Recapping MDS 2026: Biomarkers, Aging, and Prodromal Movement Disorders Marco Meglio, Odinachi Oguh, MD

Odinachi Oguh, MD, movement disorder specialist at the Cleveland Clinic Lou Ruvo Center for Brain Health, shares clinical perspectives on biomarkers, aging, non-motor symptoms, and prodromal disease from the 2026 MDS Congress.

New CTE Prevalence Estimate Underscores Push for Diagnosis Living Patients Louie Pasculli, Rory Siegel, DO, Julian Bailes, MD

A study of deceased former NFL players estimated that nearly 75% had CTE neuropathology, while a phase 3 trial is evaluating whether F-18 FNT-PET imaging could help identify disease-associated patterns in living patients.

Consistent TAPS Use Associated With Underlying Tremor Improvement in Essential Tremor Isabella Ciccone, MPH

An analysis of the PROSPECT trial reported that consistent use of transcutaneous afferent patterned stimulation was associated with improvement in underlying tremor severity in patients with essential tremor.

FDA Approves Factor XIa Inhibitor Asundexian for Secondary Stroke Prevention Marco Meglio, Isabella Ciccone, MPH

The FDA has approved asundexian, the first factor XIa inhibitor indicated to reduce recurrent stroke after a noncardioembolic ischemic stroke or high-risk TIA, based on phase 3 OCEANIC-STROKE data.

New York Forum Tackles Unmet Needs in Multiple Sclerosis Care Marco Meglio

At a NeurologyLive Clinical Forum in New York, Mary Ann Piccone, MD, of Holy Name Medical Center, led a wide-ranging discussion among MS specialists on the limits of current high-efficacy therapies, the pipeline of emerging agents, and the gap between physician and patient treatment priorities.

FDA Investigates Confirmed Insertional Oncogenesis With Spinal Muscular Atrophy Gene Therapy Isabella Ciccone, MPH

Postmarketing reports of product-related insertional oncogenesis involving GLI1 have prompted an FDA investigation into malignancy risk with SMA gene therapy, onasemnogene abeparvovec.

Todays Updates

Understanding the Long-Term Effects of Traumatic Brain Injury in Military Personnel: Clayton Mucha, MD Louie Pasculli, Clayton Mucha, MD

In honor of Veterans Day, Clayton Mucha, DO, neurorehabilitation physiatrist at Burke Rehabilitation in New York, discusses the lasting effects of traumatic brain injury in military personnel.

GV101 Gains FDA Fast Track Status for Friedreich Ataxia Ahead of Phase 2 Trial Isabella Ciccone, MPH

GV101 receives FDA fast track designation for the treatment of Friedreich ataxia, with a phase 2 trial planned to begin enrollment in late 2026.

FDA Grants Fast Track Status to FRP-003 for Long COVID Ahead of Phase 2 Study Isabella Ciccone, MPH

The FDA granted fast track designation to FRP-003, an investigational oral botanical drug candidate for Long COVID, ahead of a planned phase 2 trial evaluating fatigue in approximately 220 adults.

Headlining Current Advances and Challenges in Multiple Sclerosis Care Robert Bermel, MD, Louie Pasculli

Robert Bermel, MD, discusses advances in disease-modifying therapies, the challenges of monitoring MS progression, and emerging biomarkers and treatments that may address remaining unmet needs.

Q&A: Healthy Skin Month—What Clinicians Should Watch in Skin Cancer Care

Q&A: Healthy Skin Month—What Clinicians Should Watch in Skin Cancer Care

Todays Updates

Understanding the Long-Term Effects of Traumatic Brain Injury in Military Personnel: Clayton Mucha, MD Louie Pasculli, Clayton Mucha, MD

In honor of Veterans Day, Clayton Mucha, DO, neurorehabilitation physiatrist at Burke Rehabilitation in New York, discusses the lasting effects of traumatic brain injury in military personnel.

GV101 Gains FDA Fast Track Status for Friedreich Ataxia Ahead of Phase 2 Trial Isabella Ciccone, MPH

GV101 receives FDA fast track designation for the treatment of Friedreich ataxia, with a phase 2 trial planned to begin enrollment in late 2026.

FDA Grants Fast Track Status to FRP-003 for Long COVID Ahead of Phase 2 Study Isabella Ciccone, MPH

The FDA granted fast track designation to FRP-003, an investigational oral botanical drug candidate for Long COVID, ahead of a planned phase 2 trial evaluating fatigue in approximately 220 adults.

Headlining Current Advances and Challenges in Multiple Sclerosis Care Robert Bermel, MD, Louie Pasculli

Robert Bermel, MD, discusses advances in disease-modifying therapies, the challenges of monitoring MS progression, and emerging biomarkers and treatments that may address remaining unmet needs.

Q&A: Healthy Skin Month—What Clinicians Should Watch in Skin Cancer Care

Q&A: Healthy Skin Month—What Clinicians Should Watch in Skin Cancer Care

Latest News

Odinachi Oguh, MD, movement disorder specialist at the Cleveland Clinic Lou Ruvo Center for Brain Health, shares clinical perspectives on biomarkers, aging, non-motor symptoms, and prodromal disease from the 2026 MDS Congress.

A study of deceased former NFL players estimated that nearly 75% had CTE neuropathology, while a phase 3 trial is evaluating whether F-18 FNT-PET imaging could help identify disease-associated patterns in living patients.

An analysis of the PROSPECT trial reported that consistent use of transcutaneous afferent patterned stimulation was associated with improvement in underlying tremor severity in patients with essential tremor.

The FDA has approved asundexian, the first factor XIa inhibitor indicated to reduce recurrent stroke after a noncardioembolic ischemic stroke or high-risk TIA, based on phase 3 OCEANIC-STROKE data.

At a NeurologyLive Clinical Forum in New York, Mary Ann Piccone, MD, of Holy Name Medical Center, led a wide-ranging discussion among MS specialists on the limits of current high-efficacy therapies, the pipeline of emerging agents, and the gap between physician and patient treatment priorities.

Postmarketing reports of product-related insertional oncogenesis involving GLI1 have prompted an FDA investigation into malignancy risk with SMA gene therapy, onasemnogene abeparvovec.

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Sevasemten Misses Primary Endpoint in Pivotal Trial for Becker Muscular Dystrophy Marco Meglio

Servier reported that sevasemten did not meet its primary or secondary endpoints in the pivotal GRAND CANYON trial for Becker muscular dystrophy.

Elizabeth Spurgeon, MD, on Lifestyle Interventions as an Adjunct to Epilepsy Care Isabella Ciccone, MPH, Elizabeth Spurgeon, MD

An epileptologist at Cleveland Clinic discussed findings from a recently published study of lifestyle interventions in epilepsy and why these approaches should complement rather than replace antiseizure medications.

PREVAiLS Phase 3 Trial Evaluates Pridopidine in Early, Rapidly Progressive ALS Louie Pasculli

The 500-participant study will assess whether pridopidine, an investigational sigma-1 receptor agonist, can slow functional decline and improve survival in patients with early, rapidly progressing amyotrophic lateral sclerosis.

Distinguishing Lambert-Eaton Myasthenic Syndrome From Ocular Myasthenia Gravis in the Eye Clinic Marco Meglio

Kiril Kiprovski, MD, clinical professor of neurology and director of the Division of Neuromuscular Medicine at NYU Langone, reviews the clinical clues, bedside tests, and referral considerations that help eye care clinicians identify Lambert-Eaton myasthenic syndrome in patients with fluctuating ptosis or diplopia.

Final Results Reported for EPISOD1 Trial of SOD1-ALS Gene Therapy AMT-162 Marco Meglio

uniQure presented final safety and biomarker results from the discontinued EPISOD1 trial of AMT-162, an investigational gene therapy for SOD1-associated ALS.

Analyzing Inebilizumab’s Durable Benefit in MINT Open-Label Extension Marco Meglio

Richard Nowak, MD, director of the Yale Myasthenia Gravis Clinic and global principal investigator of the MINT trial, discusses open-label extension findings for inebilizumab, a CD19-directed B cell depleting therapy, in generalized myasthenia gravis.

IJMSC Webinar Series Guides MS Clinicians From Idea to Publication Matt Hoffman

IJMSC editor-in-chief, François Bethoux, MD, and colleagues cover idea generation, poster conversion, literature reviews, and peer review in a 4-part webinar series for MS clinicians.

FDA Grants Priority Review to Huntington Disease Gene Therapy BLA Marco Meglio

The FDA accepted uniQure's biologics license application for ifezuntirgene inilparvovec and granted it Priority Review, setting a target action date of May 1, 2027.

On Beyond Seizures: Unmet Needs in Pediatric Epilepsy Clinics Susan Duberstein, MD

Pediatric epilepsy care goes beyond seizures, addressing mental health, learning needs, coordinated referrals, equitable access, and smoother transition to adult services.

NeuroVoices: Cindy Zadikoff, MD, MSc, on the FDA Approval of Tavapadon for Parkinson Disease Isabella Ciccone, MPH, Cindy Zadikoff, MD, MSc

The executive medical director at AbbVie discussed the FDA approval of tavapadon, the first D1/D5 dopamine agonist for Parkinson disease, including the supporting TEMPO trial data and safety considerations.

Alzheimer Agent Sabirnetug Misses Primary Endpoint, Acumen Halts Development Marco Meglio

Sabirnetug failed to meet its primary endpoint in Acumen Pharmaceuticals' phase 2 ALTITUDE-AD trial for early Alzheimer disease, prompting the company to end development.

Brezosiran Demonstrates Mutant Huntingtin Reduction in Phase 1b Huntington Disease Study Louie Pasculli

Alnylam Pharmaceuticals reported initial Phase 1b findings showing brezosiran reduced cerebrospinal fluid mutant huntingtin levels by a mean of 60% to 64% at 3 months, with plans for a pivotal study in 2027.

AL-S Pharma Details Planned Phase 3 Trial Design for AP-101 in ALS Marco Meglio

AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

This Week on NeurologyLive® — November 2, 2026 Isabella Ciccone, MPH

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

Benitec Reports Positive Interim Results for BB-301 in Oculopharyngeal Muscular Dsytrophy Marco Meglio

Interim phase 1b/2a data showed all patients treated with high- or low-dose BB-301 for oculopharyngeal muscular dystrophy met formal response criteria.

Zola-Cel and Treatment-Free Remission: What the BREAKFREE-2 Data Show Marco Meglio, Jeffrey Cohen, MD

Jeffrey Cohen, MD, professor of neurology and staff physician at the Cleveland Clinic Mellen Center for Multiple Sclerosis, discusses interim BREAKFREE-2 findings for zola-cel, a CD19-directed CAR T cell therapy, in relapsing and progressive MS.

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