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

Published Date: 12 Nov 2026

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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FDA Action Update, October 2026: Approval, Acceptance, and Designations Isabella Ciccone, MPH

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NeurologyLive® Brain Games: November 8, 2026 Louie Pasculli

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ALS Agent Dazucorilant Associated With Lower 2-Year Mortality in Exploratory DAZALS Analysis Isabella Ciccone, MPH

Two-year data from the phase 2 DAZALS trial showed a lower risk of death with dazucorilant 300 mg/day vs placebo in ALS, although the study did not meet its functional primary end point.

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Closing the COVID-19 Prevention Gap This Flu Season, With Juanita Mora, MD

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Examining High-Dose Influenza Vaccination and Alzheimer Disease Risk Louie Pasculli

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Todays Updates

Closing the COVID-19 Prevention Gap This Flu Season, With Juanita Mora, MD

Closing the COVID-19 Prevention Gap This Flu Season, With Juanita Mora, MD

Examining High-Dose Influenza Vaccination and Alzheimer Disease Risk Louie Pasculli

Darren Gitelman, MD, senior medical director of the Advocate Memory Center in Park Ridge, discusses findings linking high-dose influenza vaccination to lower Alzheimer disease risk, study limitations, and potential implications for future dementia prevention research.

Latest News

Interim phase 1b data showed cizutamig produced rapid, sustained improvement in generalized myasthenia gravis with no cases of cytokine release syndrome.

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

Williams, MD, is joined by Marcus Gates, MD, and they discuss recognizing cervical nerve root and spinal cord compression, navigating treatment options, and determining when surgery may be appropriate.

Newly presented biomarker and clinical findings from the phase 2b PARADIGM trial of PrimeC informed the design of the global phase 3 PARAGON trial in ALS.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the 2026 AHA/ASA Guideline for Adult Stroke Rehabilitation and Recovery!

Two-year data from the phase 2 DAZALS trial showed a lower risk of death with dazucorilant 300 mg/day vs placebo in ALS, although the study did not meet its functional primary end point.

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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.

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.

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.

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.

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.

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.

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.

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

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

Understanding Advances Shaping Precision Medicine for Generalized Myasthenia Gravis Marco Meglio

Shaida Khan, DO, associate professor of neurology at UT Southwestern Medical Center, reflected on a recent NeurologyLive Clinical Forum on generalized myasthenia gravis and discussed how FcRn and complement inhibitors are reshaping treatment algorithms for the disease.

NeurologyLive® Friday 5 — October 30, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 30, 2026.

Distinguishing Lambert-Eaton Myasthenic Syndrome From Myasthenia Gravis: Ocular and Systemic Clues Isabella Ciccone, MPH

Although ocular signs in LEMS are typically mild and appear late, recognizing them alongside proximal weakness and autonomic symptoms can speed diagnosis and earlier detection of an associated lung cancer.

Examining Trontinemab in Early Alzheimer Disease: The Phase 3 TRONTIER 1 and 2 Trials Isabella Ciccone, MPH

The phase 3 TRONTIER 1 and 2 trials will test whether trontinemab's faster brain entry and rapid amyloid clearance translate into slower clinical decline in early Alzheimer disease.

Episode 178: Evolving Approaches to Epilepsy Clinical Trial Design Isabella Ciccone, MPH, M. Scott Perry, MD, Marco Meglio

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with M. Scott Perry, MD. [LISTEN TIME: 15 minutes]

Insights on the Updated 2026 AHA/ASA Stroke Rehabilitation Guidelines Louie Pasculli, Talya Fleming, MD, Sara Cuccurullo, MD

Sara Cuccurullo, MD, FAAPMR, and Talya Fleming, MD, FAAPMR, of Hackensack Meridian JFK Johnson Rehabilitation Institute, discuss the updated adult stroke rehabilitation and recovery guidelines and their implications for clinical practice.

World Stroke Day 2026: Closing the Gap Between Stroke Onset and the Emergency Call Number Isabella Ciccone, MPH, Brian Snelling, MD

Brian Snelling, MD, director of the stroke program at Marcus Neuroscience Institute discussed delays in calling 911 after stroke onset, gaps in awareness of atypical symptoms, and how to reach underserved populations.

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