Marco Meglio

Neurizon Therapeutics announced updates to the HEALEY-ALS trial and promising NUZ-001 results, signaling a major step forward in ALS treatment research.

Tolebrutinib's potential approval as the first brain-penetrant BTK inhibitor for non-relapsing secondary progressive MS and to slow disability accumulation independent of relapse activity could represent a paradigm shift in treating disability driven by smoldering neuroinflammation.

Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.

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 MDA Clinical & Scientific conference.

The phase 2 CANYON trial findings presented at the 2025 MDA conference highlight sevasemten’s potential in reducing muscle injury biomarkers in Becker muscular dystrophy.

The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Robert Califf, MD. [LISTEN TIME: 9 minutes]

The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, shared his powerful journey as a patient advocate and gene therapy pioneer, shedding light on the challenges and progress in LGMD.

Two separate long-term extension studies met their pre-specified endpoints, showing highly significant evidence of sustained treatment benefits in slowing disease progression in pediatric and adult patients.

The chief scientific officer at Satellos Bioscience gave clinical insight on a new novel treatment and its mechanism of action in treating Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

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

The chief scientific officer at Avidity Biosciences provided clinical perspective on the function and mechanism of del-zota, an investigational antisense treatment in development for DMD amenable to exon 44 skipping. [WATCH TIME: 3 minutes]

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.

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.

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.

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.

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]

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

Treatment with the Chordate System led to a significantly greater reduction in monthly headache days during the performance assessment period, with a reduction of 3.5 days compared to 1.2 days in the sham group.

Daniel Ontaneda, MD, PhD, a professor of neurology at the Cleveland Clinic Lerner College of Medicine, gave thoughts and perspectives on some of the emerging, promising advances in multiple sclerosis care expected to come to fruition in 2025.

PAP therapy significantly improved cognitive function, particularly executive and psychomotor skills, in patients with Parkinson disease with comorbid obstructive sleep apnea.