Marco Meglio

Brad Chapman, head of U.S Epilepsy and Rare Syndromes at UCB, provided context on some of the innovative research and studies being presented at the 2024 American Epilepsy Society Annual Meeting.

In both the randomized controlled trial and open-label extension, a higher proportion of patients without VNS achieved at least 50% and 75% seizure reductions compared to those with concomitant VNS treatment.

Fenfluramine showed a consistent safety profile in treating Lennox-Gastaut syndrome, with common adverse events such as decreased appetite, fatigue, and somnolence.

Although the study was limited, the data warrant further research and a raised awareness of the elevated risk associated with sleep apnea comorbidities in younger patients with epilepsy.

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Participants in the intervention group also reported themes of increased connectivity, creativity, and positivity.

Treatment with relutrigine resulted in a 46% placebo-adjusted reduction in motor seizures, along with a well-tolerated safety profile with adverse events that were mild to moderate.

Treatment with bexicaserin, a highly selective 5-HT2c receptor superagonist, resulted in reduction in countable motor seizures in patients not previously exposed to the agent.

CBD maintained a favorable safety profile, with 63% of participants experiencing adverse events such as diarrhea, vomiting, decreased appetite, and lethargy, leading to discontinuation in four patients.

RGX-202 demonstrated improved functional outcomes and robust microdystrophin expression, with levels exceeding those of other approved or investigational gene therapies.

Stoke Therapeutics is preparing for a phase 3 registrational study to further assess STK-001, with results from open-label extensions showing sustained seizure reductions and a well-tolerated safety profile.

Perspective from neurologists on the current and future applications for artificial intelligence across a bevy of neurologic conditions.

Early data from the pilot RELIEV-CM trial showed significant reductions in headache and migraine days, with 56% of patients showing a 50% reduction in symptoms at 4 weeks, and 100% at 12 weeks.

The professor of neurology at the University of Alabama at Birmingham Epilepsy Center commented on various topics related to infantile spasms amid Infantile Spasms Awareness Week.

Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at Wake Forest School of Medicine, discussed a new, innovative trial assessing the therapeutic potential of self-administered subcutaneous rozanolixizumab as a treatment for myasthenia gravis.

QRL-201, developed by QurAlis, aims to restore STATHMIN-2 (STMN2) expression, a key protein for neural repair, which is significantly reduced in patients with ALS.

To date, BT-267 shows strong potential as a best-in-class LRRK2 inhibitor for Parkinson disease, with a superior safety profile, excellent pharmacokinetics, and innovative development leveraging advanced technologies and biomarker modeling.

Delaying treatment past 12 months worsens leg disability scores and leads to a more challenging disease course in CIDP patients.

The launch of lorcaserin studies in 2020 coincided with several patients starting the newly available fenfluramine, which may have contributed to challenges in enrolling enough participants.

The application is based on a large-scale phase 3 study in which blarcamesine demonstrated a significant decline in disease progression compared with placebo over a 48-week period.

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Subgroup analyses revealed stronger efficacy signals in biomarker-defined groups, particularly in Caucasian participants with measurable baseline myostatin levels.

These results highlight the age range and ambulation status for which viltolarsen is a key treatment option for DMD patients amenable to exon 53 skipping.

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Applied Therapeutics noted that it is reviewing the FDA's feedback and will request a meeting to discuss requirements for either a resubmission of the NDA or appeal of the agency's decision.

Over the 6-month treatment period, patients saw improvements in cataplexy frequency, excessive daytime sleepiness, cognition, and work productivity with AXS-12.

AL002 did not meet the primary endpoint of slowing Alzheimer's clinical progression, as measured by the Clinical Dementia Rating Sum of Boxes and showed no treatment effects on secondary clinical and functional endpoints.

Over 1 year of treatment, simufilam failed to distinguish itself from placebo on the primary end points of Alzheimer’s Disease Assessment Scale-Cognitive Subscale 12 and Alzheimer’s Disease Cooperative Study-Activities of Daily Living.

In a 76-patient cohort, HL192 was safe and well tolerated across 5 observed dose levels, with a treatment-emergent adverse event incidence that mirrored placebo.

The study, presented at the 2024 AANEM) Annual Meeting, evaluated 52 patients who underwent surgery for carpal tunnel syndrome and explored the comparative effectiveness of the Conway and Zeidman scales in predicting patient outcomes.