NeurologyLive

01/16/2025

Barry Ticho, MD, PhD, chief medical officer at Stoke Therapeutics discussed recent developments for zorevunersen, including the significance of breakthrough therapy designation and its plan for a phase 3 trial.

The chief medical officer at Stoke Therapeutics discussed recent developments for zorevunersen, including the significance of breakthrough therapy designation and its plan for a phase 3 trial. [WATCH TIME: 5 minutes]

01/16/2025

Bayer AG and BlueRock Therapeutics have unveiled new details into a phase 3 registrational trial dubbed exPDite-2 that will assess the efficacy and safety of bemdaneprocel, an investigational cell therapy that replaces lost dopaminergic producing neurons, in patients with Parkinson disease. The study, a first-of-its-kind in the field, is anticipated to begin in the first half of 2025 and depending on the outcome, are intended to be a part of a robust data package for future regulatory submissions.

The newly announced sham surgery-controlled trial will include 102 patients with moderate PD testing several end points, such as change in ON-time without troublesome dyskinesia, over a 78-week period.

01/16/2025

According to a new announcement from Medtronic, the company has gained CE Mark approval in the European Union and the United Kingdom for its BrainSense Adaptive deep brain stimulation and BrainSense Electrode Identifier, 2 technologies that will help contribute more efficient, patient-specific care for individuals with Parkinson disease.

Adaptive DBS dynamically adjusts stimulation based on patient brain activity, improving therapy customization and motor symptom management in real-time.

01/15/2025

New data from the phase 1 PET and MAD-2 trials of RAP-219, a therapeutic in development for focal , showed that treatment with the agent was generally well tolerated, with target exposures and receptor occupancy achieved within 5 days of dosing.

An ongoing phase 2a study of RAP-219 involves adult patients with drug-resistant epilepsy who have an implanted responsive neurostimulation (RNS) device, allowing for real-time monitoring of intracranial EEG data.

01/15/2025

At , Christina J. Azevedo, MD, of Keck School of Medicine of USC, talked about recent research that highlights the preclinical phase of . Check out the full conversation on our site now! European Committee for Treatment & Research in Multiple Sclerosis

01/15/2025

New data from an open-label extension of the phase 2/3 AD-004 trial showed that treatment with blarcamesine resulted in continued treatment benefits through up to 4 years in patients with early-stage disease. Anavex Life Sciences Corp.

Using a delayed-start analysis, treatment differences on outcomes of ADAS-Cog13 and ADCS-ADL continued to grow larger during weeks 144 and 192 of the open-label extension.

01/15/2025

According to a recent announcement, Promis Neurosciences’ phase 1b PRECISE-AD trial of PMN310, a monoclonal antibody in development for patients with disease, is now underway

Eligible participants are ambulatory adults aged 50 or older with mild cognitive impairment or mild Alzheimer disease, confirmed by clinical assessments and a positive amyloid PET scan, and who meet specific health and safety criteria.

01/15/2025

Originally published by Contagion Live a recent study led by Or Shemesh, PhD, of University of Pittsburgh, revealed a significant association between ICP27, a protein produced by herpes simplex virus 1, and the severity of disease. 🧠 🦠

Recent research investigated how HSV-1 infection affects tau phosphorylation through the cGAS-STING-TBK1 pathway, highlighting potential new therapeutic strategies for Alzheimer disease.

01/15/2025

🗣️🎙️We recently had a conversation with Josef Coresh, MD, PhD, of NYU Grossman School of Medicine, who talked about a recent study that estimated the lifetime risk of . To learn more, view the interview on our site now! 🎥🧠 NYU Langone Health

01/14/2025

At , James Wheless, MD, of the University of Tennessee Health Science Center, sat down to discuss the optimization and advantages of stiripentol, an FDA-approved treatment for patients with Dravet syndrome.

More: https://www.neurologylive.com/view/optimizing-epilepsy-treatments-stiripentol-role-dravet-syndrome

01/14/2025

Promis Neurosciences' phase 1b PRECISE-AD trial (NCT06750432), a randomized, double-blind, placebo-controlled trial of PMN310, a monoclonal antibody in development for patients with Alzheimer disease (AD), is now underway. PMN310, a humanized IgG1 treatment targeting toxic amyloid-ß (Aß) oligomers, aims to reduce AD pathology while minimizing risk of amyloid-related imaging abnormalities (ARIA) linked with existing approved therapies.

Eligible participants are ambulatory adults aged 50 or older with mild cognitive impairment or mild Alzheimer disease, confirmed by clinical assessments and a positive amyloid PET scan, and who meet specific health and safety criteria.

01/13/2025

Do you manage patients with NF1-PN? Register today for our national broadcast with Drs. Rosser & Nghiemphu. Listen to experts discuss challenges with current management options, the challenges for adult patients with NF1-PN and the transition from pediatric care. bit.ly/3zu9MVX

01/13/2025

In this Peers & Perspectives episode, panelists Gabriel Pardo, MD, and Martin Belkin, DO, discuss how in multiple sclerosis (MS) treatment, clinicians must strategically evaluate disease progression, considering within-class or mechanism-of-action switches.

Panelists discuss how in multiple sclerosis (MS) treatment, clinicians must strategically evaluate disease progression, considering within-class or mechanism-of-action switches. Comprehensive monitoring, personalized approaches, and proactive management are crucial for optimizing patient outcomes an...

01/13/2025

Top stories in from this week include:

🔺 Anti-tau therapy posdinemab gains FDA Fast Track designation
🔺 Gene therapy SGT-212 to enter Friedreich ataxia trial
🔺 Zorevunersen eyes phase 3 study of Dravet syndrome

Neurology News Network. for the week ending January 11, 2025. [WATCH TIME: 4 minutes]

01/13/2025

A new phase 1b/2a proof-of-concept trial (NCT06705192), dubbed SORT-IN-2, will assess the therapeutic potential of investigational VES001 as a potentially disease-modifying therapy for patients with mutations in their progranulin-coding gene (GRN), the root cause for frontotemporal dementia (FTD). The trial, initiated in recent days, is expected to complete dosing for all participants by mid-2025.

The SORT-IN-2 trial is a Phase 1b/2a proof-of-concept study evaluating the therapeutic potential of VES001, a novel oral treatment designed to target GRN mutations, which are the root cause of frontotemporal dementia.

01/13/2025

Shaping out to be an amazing year for conference coverage! In this article, we give our clinical audience insights on which conferences we will be attending in 2025, highlighting the great advances in the field🥼🧬🔬

https://www.neurologylive.com/view/neurologylive-mapping-out-our-2025-conference-journey

01/12/2025

In a recent interview, Nigel Bunnett, PhD, BSc, of NYU Langone Health, provided a clinical overview of preclinical research identifying a new receptor for nerve growth factor that plays an important role in pain signaling.

https://www.neurologylive.com/view/discovering-new-receptor-nerve-growth-factor-nigel-bunnett

01/12/2025

In recent news, the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Denali Therapeutics’ investigational therapy tividenofusp alfa, also known as DNL310, for the treatment of patients with .

Results from the open-label phase 1/2 study of tividenofusp alfa demonstrated positive effects on evidence-based surrogate end points in participants with Hunter syndrome.