Marco Meglio

James Wheless, MD, FAAP, FAAN, FAES, Le Bonheur Chair in Pediatric Neurology at the University of Tennessee Health Science Center, shared insights on his presentation from AES 2024, focusing on stiripentol, an FDA-approved treatment for Dravet syndrome.

Using a delayed-start analysis, treatment differences on outcomes of ADAS-Cog13 and ADCS-ADL continued to grow larger during weeks 144 and 192 of the open-label extension.

Eligible participants are ambulatory adults aged 50 or older with mild cognitive impairment or mild Alzheimer disease, confirmed by clinical assessments and a positive amyloid PET scan, and who meet specific health and safety criteria.

An ongoing phase 2a study of RAP-219 involves adult patients with drug-resistant epilepsy who have an implanted responsive neurostimulation (RNS) device, allowing for real-time monitoring of intracranial EEG data.

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 restless legs syndrome.

The SORT-IN-2 trial is a Phase 1b/2a proof-of-concept study evaluating the therapeutic potential of VES001, a novel oral treatment designed to target GRN mutations, which are the root cause of frontotemporal dementia.

Between those infected and uninfected with COVID-19, investigators observed no differences in symptom trajectories, assessed through SymptoMScreen.

The senior vice president of research at The ALS Association discussed the need for standardizing ALS diagnosis across institutions to reduce delays and ensure timely treatment. [WATCH TIME: 4 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Russell Lebovitz, MD, PhD. [LISTEN TIME: 22 minutes]

Masoud Toloue, chief executive officer at Quanterix, and Nicholas J. Ashton, PhD, senior director of the Banner Health Fluid Biomarker Program, discussed the Simoa technology required to test for p-tau217, the expanding understanding of this biomarker, and the next steps for advancing this research.

Posdinemab, a monoclonal antibody that targets the mid-domain of Alzheimer disease-specific phosphorylated tau, is currently being investigated in a placebo-controlled phase 2b trial that employs a plasma biomarker as a screening tool.

The revised protocol merges the 6-month symptomatic and 18-month disease-modifying studies into a single trial with a 6-month symptomatic readout and a 12-month disease-modifying evaluation.

SGT-212 delivers full-length frataxin via MRI-guided intradentate nucleus infusion and intravenous administration to target mitochondrial dysfunction in neurons and cardiomyocytes, addressing both neurologic and cardiac symptoms.

The global Phase 3 trial aims to enroll ~150 patients with Dravet syndrome who have SCN1A variants, assessing zorevunersen's impact on seizure frequency, behavior, cognition, and safety over 60 weeks, with results expected by 2027.

Lekha Rao, MD, an epileptologist at UCLA Health, discussed the challenges in diagnosing D/EE-SWAS, the importance of early EEG, and the need for greater awareness around this rare and complex epileptic condition.

Despite not meeting its primary and key secondary end points, an exploratory high dose cohort of fosigotifator demonstrated promising results on end points of muscle strength.

Over a 24-week period, DNL343 failed to distinguish itself from placebo on the primary end point of ALSFRS-R, as well as on other secondary end points of muscle strength and respiratory function.

Babitha Haridas, MD, MBBS, a pediatric epileptologist at Johns Hopkins Medicine, discussed the complexities with managing status epilepticus in Lennox-Gastaut syndrome, focusing on the importance of identifying triggers early in their condition.

Most participants showed improvements in motor function, with 61% achieving a clinically meaningful increase in HFMSE scores by week 52 and 41% achieving similar gains in RULM scores.

The ACCORD-2 trial demonstrated a significant reduction in overall worsening compared to placebo, while the ADVANCE-2 trial showed numerically greater improvements without reaching statistical significance.

Ultimately, the biomarkers' temporal patterns may help differentiate attacks from remission, with sGFAP being particularly useful in distinguishing genuine attacks from pseudoattacks.

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 vascular dementia.

David Lynch, MD, PhD, a professor of neurology at the University of Pennsylvania Perelman School of Medicine, discussed the significance of a number of recently presented analyses highlighting omaveloxlone, the first approved therapy for Friedreich ataxia.

Imlifidase-IVIg treatment led to faster disability improvement (3 weeks) and earlier independent walking (6 weeks) compared to IVIg alone.

Novartis plans to present OAV101 IT data at a 2025 medical meeting and share results with regulatory agencies, including the FDA, to support approval for patients with SMA in need.

Mind Moments®, a podcast from NeurologyLive®, brings you a review of 2024, with insights from Daniel Ontaneda, MD, PhD; Ian Kremer; Andy Berkowski, MD, PhD; Sameea Husain-Wilson, DO; Jonathan Parker, MD, PhD; and Lawrence Robinson, MD. [LISTEN TIME: 25 minutes]

The complement system is critical in immune defense and tissue homeostasis, but its dysregulation can contribute to autoimmune neurological disorders and neurodegenerative diseases like Alzheimer, ALS, and multiple sclerosis.

Vatiquinone, a small molecule inhibitor, demonstrated its efficacy and safety across a range of age groups with Friedreich ataxia, with effects seen on disease progression and specific subscales of the mFARS.

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 CGRP medications to treat migraine.

Human studies revealed thinner myelin sheaths in CD59-deficient patients, indicative of a process of segmental demyelination followed by remyelination.