Overall, vatiquinone-treated patients demonstrated persistent effects on disease progression over a 144-week long-term period, leading to a future regulatory submission.

RAG-18 is an saRNA delivered to muscle tissue to increase UTRN expression and compensate for DMD defects regardless of the location of mutation on DMD gene.

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for U.S. and EU regulatory submissions in Q1 2025.

Michelle Bravo, MD, an assistant professor of clinical neurology at the University of Miami, provided commentary on a rare case report of a subdural hematoma linked to spontaneous intracranial hypotension caused by a refractory CSF leak.

Over half of the patients (56.7%) discontinued at least one class of acute migraine medication within 12 months post-erenumab initiation.

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

The orphan medicinal product designation given by the European Commission was based on findings produced in research funded by the United States Department of Defense.

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 insomnia.

Ongoing analysis of vamorolone's effects as a mineralocorticoid receptor antagonist in healthy adults is underway, with results to be presented at upcoming medical conferences.

The phase 2 study, expected to include 575 patients with acute migraine, will use percentage of participants with freedom from pain and freedom from the most bothersome symptom as the coprimary end points.

Over a 12-month period, patients showed sustained improvements in disease progression through valbenazine, regardless of concomitant antipsychotic treatment.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 4, 2024.

Amifampridine enhances neuromuscular transmission and relieves muscle weakness, showing promise for Lambert-Eaton myasthenic syndrome and other neuromuscular junction disorders.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Daniel Ontaneda, MD, PhD. [LISTEN TIME: 27 minutes]

Sleep is critical for physical and mental health, with its insufficiency leading to various disorders and increased health risks; thus, clinicians should integrate sleep assessments and hygiene strategies into their clinical care.

The KINETIC 2 study did not show statistically significant improvement in essential tremor symptoms compared to placebo, leading to the termination of its development for this condition.

Transition to college life poses unique challenges for students living with migraine, and a comprehensive checklist helps ensure they are prepared for this critical phase.

The Total Posterior Spine System offers a promising alternative to spinal fusion, preserving mobility and reducing strain on adjacent spinal levels in patients with spondylolisthesis and spinal stenosis.

This trial aims to explore the role of the noradrenergic system in behavioral and cognitive outcomes and could lead to new therapeutic strategies for managing PSP.

ION464 is designed to inhibit the production of the alpha-synuclein protein, aiming to reduce the accumulation of this protein in the brain, which is believed to be a key factor driving neurodegeneration in MSA and Parkinson disease.

The consultant neurologist and senior lecturer at St. Georges Hospital in London, provided commentary on the diagnosis, management, and future research around neuropsychiatric symptoms of Parkinson disease.

A recent survey showed that patients with Parkinson disease quickly adapted to using continuous subcutaneous apomorphine infusion therapy, with high amount of recommending the device to others.

Amantadine extended-release capsules, approved for levodopa-related dyskinesia and OFF episodes in Parkinson disease, offer high amantadine concentrations that peak in the time of first morning levodopa dose.

Nina Riggins, MD, PhD, FAAN, FAHS, UCNS Diplomate, highlighted the essential role of advocacy in advancing neurological care, particularly in improving the transition process for pediatric patients with migraine to adult care.

ELATE, a multicenter, randomized, double-blind, placebo-controlled trial, aims to address the limitations of current oral therapies by exploring a targeted, localized treatment option.