Real-world findings from the RESTORE registry presented at the 2025 MDA conference confirmed the established safety profile of onasemnogene abeparvovec over a 5-year period.

Patient advocate and MDA Ambassador Lily Sander shared inspiring perspective on Charcot-Marie-Tooth disease, advocacy efforts, and the importance of connection

Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.

Alpha Cognition has officially launched its new treatment for mild to moderate Alzheimer disease, with a focus on expanding patient access in the long-term care market.

Findings from the phase 2 FIGHT DMD trial suggest an investigational oral thromboxane prostanoid receptor antagonist may preserve heart function in patients with DMD-associated cardiomyopathy.

This phase 4 study on omaveloxolone will assess long-term maternal and infant health, potentially informing future safety guidelines for patients with Friedreich ataxia.

The MyacarinG study post-hoc analysis demonstrated rozanolixizumab's potential to significantly reduce ocular symptoms in gMG, with consistent improvements across multiple scoring systems.

Three patients treated with DT-DEC01 showed improvements in various functional tests, including echocardiography, arm movements, grip strength, and spirometry after 12-24 months of treatment.

In the study, most participants are aged 18-60, with 53% female and 73.1% carrying the c.826C>A mutation, while 15 patients are aged 12-18.

A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.

Spanning 14 weeks, the study evaluates age-appropriate dosing, safety, pharmacokinetics, and clinical effects through once-weekly injections of efgartigimod PH20 SC in juvenile myasthenia gravis.

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

Over the 5-year period, treatment-related adverse events with the gene therapy were mild or moderate, with most occurring within the first 90 days after infusion.

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 gene therapy for neuromuscular disorders.

Erenumab (Aimovig) effectively reduced chronic headaches in a patient with a history of aneurysmal subarachnoid hemorrhage, showcasing the therapeutic potential of CGRP-targeting treatments for post-aSAH headaches.

On World Sleep Day, the president of the American Academy of Sleep Medicine discussed the importance of sleep for health, the risks of sleep deprivation, nonpharmacologic strategies to improve sleep, and the need for greater awareness of sleep disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 14, 2025.

Anne Marie Morse, DO, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children’s Hospital, dove into the importance of sleep health, its impact on brain function, and emerging research in honor of World Sleep Day.

While effective in reducing amyloid plaques, donanemab carries a notable risk of amyloid-related imaging abnormalities (ARIA), underscoring the need for careful patient monitoring and risk management strategies.

Findings from a recently published large-scale analysis highlighted the high burden of cognitive impairment among veterans with both migraine and TBI.

New research highlights the significant economic burden of misdiagnosed migraine patients, revealing higher healthcare costs and resource utilization over time compared to those with accurate diagnoses.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Bijoy Menon, MD, MSc, FRCPC. [LISTEN TIME: 18 minutes]

Participants who received the new batch of capsules during the extension phase experienced a lower incidence of falls compared with those taking the old capsules or placebo.

Explore the key 2024 neurology guideline updates shaping clinical care across stroke, epilepsy, movement disorders, and more in this essential feature.

The professor of neurology at Northwestern University Feinberg School of Medicine discussed the NSD-ISS and its potential to refine PD diagnosis and clinical trial selection through biomarker-driven staging.