Exploring the role of T cells in Alzheimer's disease, suggests new treatment strategies

Published Date: 10 Mar 2023

Nearly two dozen clinical trials targeting the immune system are in clinical trials for Alzheimer's disease.

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Phase 3b STRENGTH Trial Provides Greater Support for Intrathecal SMA Gene Therapy Marco Meglio

Phase 3b study revealed Itvisma's safety and efficacy in treating spinal muscular atrophy, showing modest motor function improvements in patients previously on other therapies.

NeuroVoices: Alireza Atri, MD, PhD, on Notable CTAD Trial Presentations, EVOKE Data Alireza Atri, MD, PhD, Isabella Ciccone, MPH

The director of the Banner Sun Health Research Institute discussed a variety of meaningful Alzheimer disease research presented at the 2024 Clinical Trials on Alzheimer's Disease Conference (CTAD).

What the First Intrathecal Gene Therapy Means for SMA Clinicians and Researchers Marco Meglio

Angela Lek, PhD, chief research officer of the Muscular Dystrophy Association, discusses the FDA approval of intrathecal onasemnogene abeparvovec (Itvisma; Novartis) and what it means for SMA treatment across older pediatric and adult populations.

Genetic Testing in Adult Epilepsy: GEAN’s Plan to Standardize Practice and Study Adult Outcomes Marco Meglio

Elizabeth Gerard, MD, an epileptologist at Northwestern Medicine, provided clinical insights on a new consortium, GEAN, which aims to expand genetic testing and define adult phenotypes for rare genetic epilepsies across North America.

A Clinical Review of the 2025 Restless Leg Syndrome Guidelines Louie Pasculli, Andy Berkowski, MD, PhD

Andy Berkowski, MD, PhD, certified sleep neurologist and author of the RLS guidelines, spoke about the clinical impact of the recently published restless syndrome guidelines.

2024 Primary Stroke Prevention Guidelines: Key Takeaways and Clinical Implications Walter Kernan, MD, Isabella Ciccone, MPH

Walter Kernan, MD, senior research scientist at Yale University, discussed the latest primary stroke prevention guidelines and their focus on lifelong brain health and risk factor management.

Todays Updates

FDA Expands Indication for n‐Butyl Cyanoacrylate in Chronic Subdural Hematoma Isabella Ciccone, MPH

The expanded indication brings n-butyl cyanoacrylate’s established performance into the treatment of chronic subdural hematoma, a condition for which traditional surgical interventions may not always be suitable or effective.

Impacts of the Lancet Commission on Obesity Treatment, With Pooja Singhal, MD

Impacts of the Lancet Commission on Obesity Treatment, With Pooja Singhal, MD

Todays Updates

FDA Expands Indication for n‐Butyl Cyanoacrylate in Chronic Subdural Hematoma Isabella Ciccone, MPH

The expanded indication brings n-butyl cyanoacrylate’s established performance into the treatment of chronic subdural hematoma, a condition for which traditional surgical interventions may not always be suitable or effective.

Impacts of the Lancet Commission on Obesity Treatment, With Pooja Singhal, MD

Impacts of the Lancet Commission on Obesity Treatment, With Pooja Singhal, MD

Latest News

Phase 3b study revealed Itvisma's safety and efficacy in treating spinal muscular atrophy, showing modest motor function improvements in patients previously on other therapies.

The director of the Banner Sun Health Research Institute discussed a variety of meaningful Alzheimer disease research presented at the 2024 Clinical Trials on Alzheimer's Disease Conference (CTAD).

Angela Lek, PhD, chief research officer of the Muscular Dystrophy Association, discusses the FDA approval of intrathecal onasemnogene abeparvovec (Itvisma; Novartis) and what it means for SMA treatment across older pediatric and adult populations.

Elizabeth Gerard, MD, an epileptologist at Northwestern Medicine, provided clinical insights on a new consortium, GEAN, which aims to expand genetic testing and define adult phenotypes for rare genetic epilepsies across North America.

Andy Berkowski, MD, PhD, certified sleep neurologist and author of the RLS guidelines, spoke about the clinical impact of the recently published restless syndrome guidelines.

Walter Kernan, MD, senior research scientist at Yale University, discussed the latest primary stroke prevention guidelines and their focus on lifelong brain health and risk factor management.

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FDA Approves Inebilizumab for AChR- and MuSK-Positive Generalized Myasthenia Gravis Isabella Ciccone, MPH

Inebilizumab is the first CD19-targeted B-cell therapy approved for antiAChR- or antiMuSK antibody–positive generalized myasthenia gravis, providing symptom control with twice-yearly dosing.

FDA Clears Pivotal Phase 3 PREVAiLS Study of Pridopidine in Early, Rapidly Progressive ALS Isabella Ciccone, MPH

The phase 3 study aims to confirm findings from the HEALEY ALS platform trial in patients with early-stage rapidly progressive ALS, which suggested potential benefits of pridopidine across multiple domains.

EPX-100 Shows Promising Seizure Reduction in Ongoing Open-Label Extension Louie Pasculli

New data from the open-label extension of Harmony Biosciences’ Phase 3 ARGUS trial show that EPX-100 (clemizole) achieved a median 50% reduction in countable motor seizures in patients with Dravet Syndrome.

Cell Therapy Deramiocel Meets Primary End Point in Phase 3 DMD Cardiomyopathy Trial Isabella Ciccone, MPH

Capricor Therapeutics anticipates that detailed phase 3 data from the HOPE-3 study will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.

Michael Sperling, MD, on AES Lifetime Achievement Award, Evolution in Epilepsy Care Michael R. Sperling, MD, Louie Pasculli

Michael Sperling, MD, Baldwin Keyes Professor of Neurology at Vice Chair for Research at the Sidney Kimmel Medical College, spoke about the significance of receiving the Founders Award and the current state of epilepsy.

Z-Rostudirsen Meets Primary End Point in Phase 1/2 DELIVER Trial for Duchenne Muscular Dystrophy Isabella Ciccone, MPH

Long-term treatment with z-rostudirsen over 24 months showed sustained functional improvements across all end points in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

FDA Accepts NDA for Low-Sodium Oxybate TRN-257 in Narcolepsy and Idiopathic Hypersomnia Isabella Ciccone, MPH

TRN-257, a treatment in development for adults with narcolepsy and idiopathic hypersomnia, is designed to reduce daily sodium exposure relative to existing higher-sodium therapies.

NeuroVoices: Richard Rammo, MD, on How Thalamic Neuromodulation is Evolving for Drug Resistant Epilepsy Marco Meglio, Richard Rammo, MD

The neurosurgeon at Cleveland Clinic discussed the goals of the AES thalamic neuromodulation special interest group and the major clinical and research priorities shaping the future of neuromodulation for drug resistant epilepsy.

AES Poster Showcases Feasibility of Mobile App to Track Daily Self-Reporting Epilepsy Outcomes Isabella Ciccone, MPH

A new study presented at AES 2025 reported that a cognitive-behavioral intervention via mobile application for patients with epilepsy can reliably capture daily self-reported outcomes.

Long-Term Expanded Access Program Data Support Phase 3 Development of Bexicaserin in Developmental Epileptic Encephalopathies Marco Meglio

New findings reveal bexicaserin significantly reduces seizure frequency in patients with developmental and epileptic encephalopathies, supporting its advancement to phase 3 trials.

FDA Action Update, November 2025: Approvals, Clearance, and Boxed Warning Isabella Ciccone, MPH

Catch up on any of the neurology news headlines you may have missed in November 2025, compiled into 1 place by the NeurologyLive® team.

Survey identifies Fever-Related Seizure Reduction as Key Feature of CDKL5 Deficiency Disorder Louie Pasculli

A recent survey reveals fever significantly reduces seizure frequency in CDKL5 Deficiency Disorder, highlighting potential therapeutic strategies for patients.

Stiripentol Reduces Seizure Burden in Patients with Lennox-Gastaut Syndrome, Study Shows Louie Pasculli

A new phase 2 study presented at AES 2025 showed that stiripentol may reduce seizure burden in patients with Lennox-Gastaut syndrome.

Inside the 2026 MDA Conference: Keynote Insights and Evolving Neuromuscular Care Marco Meglio, Sharon Hesterlee, PhD

Sharon Hesterlee, PhD, President and CEO of the Muscular Dystrophy Association, discussed the 2026 MDA Conference, the significance of its keynote speaker, and the evolving therapeutic landscape in neuromuscular medicine.

PERC Analysis Highlights Factors Influencing Candidacy for Pediatric Drug-Resistant Epilepsy Surgery Isabella Ciccone, MPH

A new study presented at AES 2025 examined factors linked to declining surgery for pediatric drug-resistant epilepsy and highlighted variability in candidate selection across centers.

Gene Therapy ETX101 Demonstrates Significant Effects on Seizure Reduction, Neurodevelopmental Outcomes in POLARIS Phase 1/2 Program Marco Meglio

The positive interim findings research represents the first clinical data for ETX101, an investigational cell-type-selective gene therapy, and the first potential one-time disease-modifying treatment for Dravet syndrome.

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