Marco Meglio

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, an associate 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.

Using more than 100,000 claims, the 36-month cumulative risk of acute myocardial infarction and stroke was similar between erenumab and other anti-CGRP mAbs, with minimal residual bias.

Treatment strategies varied across cases, incorporating therapies like prednisolone, rituximab, and azathioprine, with overall positive outcomes and symptom improvement.

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 Parkinson disease.

Lecanemab, a monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid-ß, remains under review, with a decision expected in the coming months.

BXCL501, an orally dissolving dexmedetomidine film, showed positive results in reducing agitation symptoms in patients with Alzheimer disease during the phase 3 TRANQUILITY trial.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sonal Bhatia, MD, FACNS. [LISTEN TIME: 24 minutes]

Based on the results of PROGRESS and the previous RELIEF-DPN-1 study, the 10 mg dose of pilavapadin was selected to move forward in clinical development for diabetic peripheral neuropathic pain.

In the CAVS-MS study, 19% of participants with atypical or radiological-only presentations met the 2024 McDonald Criteria for MS, enabling earlier treatment initiation.

GFAP changes positively correlated with gadolinium-enhancing lesion volume (P = 0.18) and negatively with left ventricular volume (P = -0.19).

The vice president and senior global program lead for Hematology and Cell Therapy at BMS gave clinical commentary on encouraging phase 1 findings of CD19-XT in progressive and relapsing multiple sclerosis.

In both HERCULES and GEMINI trials, tolebrutinib reduced the risk of 6-month confirmed disability worsening, especially in participants with at least 4 baseline paramagnetic rim lesions.

With a scheduled PDUFA date of August 31, 2025, deramiocel looks to become the first marketed treatment for cardiomyopathy in DMD, one of the leading causes of death in this patient population.