News

Amber Freed, founder of SLC6A1 Connect, shared her personal journey and advocacy efforts to raise awareness for SLC6A1, a rare genetic epilepsy, in honor of Rare Disease Day.

Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shed insight on the advances and promise for treating spinal muscular atrophy, in the context of Rare Disease Day.

After 4 years of treatment, those on cladribine demonstrated a correlation between sustained health-related quality of life, specifically physical and mental health, and cognition.

The executive vice president of research at National MS Society talked about how advancements in the field of neurology are shaping new approaches to diagnosing, monitoring, and treating multiple sclerosis. [WATCH TIME: 5 minutes]

Overall, 81.6% of patients preferred ocrelizumab over other MS treatments, with 98.1% satisfied with it overall by week 48.

The executive vice president of research at National MS Society discussed the role of compartmentalized inflammation, microglial interactions, and emerging therapeutic targets in multiple sclerosis. [WATCH TIME: 4 minutes]

The study will track patients over 36 months and use T2 lesion count and the number of serious infections as primary endpoints, with secondary endpoints including adverse events, relapse rates, and the achievement of no evidence of disease activity.

The neurooncologist from the University of California Los Angeles provided clinical insight on the impact of a new treatment option for adults and children with neurofibromatosis type 1 that are not amenable to complete surgical resection. [WATCH TIME: 2 minutes]

The phase 3 PERSEUS study will assess the efficacy of Sanofi’s investigational Bruton’s tyrosine kinase inhibitor tolebrutinib compared with placebo in delaying disability progression in PPMS.

RapidAI identified 98% of LVOs compared to 74% by Viz LVO, while also correctly identifying 94% of LVO-negative cases compared to 91% for Viz.

A physician assistant specializing in Parkinson disease discussed advancements in neurology aimed at enabling personalized treatment, continuous monitoring, and improved disease management. [WATCH TIME: 4 minutes]

The pediatric neurologist provided a clinical overview of the advances and roadblocks of gene therapy in neuromuscular disorders, which will be highlighted at the 2025 MDA Clinical & Scientific Conference.

Propranolol showed a stronger protective effect against ischemic stroke compared to other beta-blockers, such as metoprolol and timolol, possibly due to its unique pharmacological profile.

Topline data of the D1AMOND study showed statistical significance between ecopipam and placebo for the primary as well as secondary efficacy end points in patients with Tourette syndrome.

ANGEL-TNK demonstrated Tenecteplase's potential to enhance microcirculation post-thrombectomy, reducing brain tissue damage from inadequate blood flow.

Initial data reported from the FREEDOM-DM1 trial suggest PGN-EDODM1 has dose-dependent effects in patients with DM1, with further results from study cohorts expected in 2025 and 2026.

The John David Eaton Chair in Multiple Sclerosis at the University of Toronto gave clinical insights on emerging, innovative tracks at the 2025 ACTRIMS Forum and how these sessions align with recent trends in the multiple sclerosis field. [WATCH TIME: 4 minutes]

Over a 3-month period, galcanezumab treatment reduced definite dizzy days from 17.9 to 6.6, compared to 18.0 to 12.5 in the placebo group

The associate professor in pain management and emergency medicine at UC San Diego shared her reaction to the FDA approval of suzetrigine, a selective, nonaddictive alternative to opioids for acute pain management. [WATCH TIME: 4 minutes]

Entrada Therapeutics has recently received FDA clearance to move forward with a clinical trial for its investigational Duchenne muscular dystrophy therapy.

The approval was based on data from the ADAPT-PD trial, a prospective study that laid the foundation for aDBS use in a real-world clinical environment.

Symbravo showed a statistically significant greater migraine treatment response compared to prior oral CGRP inhibitors (P <0.001), with 47.9% of patients reporting 2-hour pain freedom for most attacks versus 1.0% with oral CGRPs

The associate professor of neurology at Columbia University provided commentary on the rapid advancements in translating genetic research into ALS treatments and the challenges that remain. [WATCH TIME: 5 minutes]

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

A recent analysis identified potential safety concerns associated with pitolisant, sodium oxybate, solriamfetol, and modafinil, highlighting distinct adverse event profiles for each narcolepsy treatment.

The director of the Cerebrovascular Center at Mount Sinai discussed how results from the ESCAPE-MeVO trial highlight the impact of anesthesia rates, thrombectomy techniques, and the need for rigorous clinical trials to minimize bias. [WATCH TIME: 3 minutes]

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 on the history of the ACTRIMS Forum.

Normotensive patients with narcolepsy on sodium oxybate had over 50% of a higher risk of new-onset hypertension or antihypertensive medication use compared with nonusers of the treatment.

Neurology News Network. for the week ending February 23, 2025. [WATCH TIME: 3 minutes]