Marco Meglio

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.

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 parasomnias.

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.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Andy Berkowski, MD, PhD. [LISTEN TIME: 26 minutes]

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.

Hearing loss, as defined by objective audiograms, was associated with an additional risk of developing PD later in life in a dose-dependent manner.

Amanda Hare, DNP, a doctor of nursing practice specialized in movement disorders, gave comments on the innovation behind the StrivePD app and how it can revolutionize personalized treatment management for patients with Parkinson disease.

The multicenter, 24-week study will feature 20 patients with MG, testing changes on MG-ADL as well as several other secondary outcomes, including patient-reported assessments.

Overall, subcutaneous efgartigimod was safe and effective in patients with seropositive MG, demonstrated by consistent improvements in Myasthenia Gravis-Activites of Daily Living.

The trial, a 20-week study assessing lamotrigine in DLB, will use change in Clinical Dementia Rating-Sum of Boxes as the primary efficacy end point, with several other notable secondary outcomes.

For more than a 2-year treatment period, 95% of patients reported taking at least 95% of their daily medications with zilucoplan.

Over a 12-month treatment period, patients on deferiprone demonstrated decreased blood ferritin and hippocampal QSM but caused accelerated cognitive decline and increased regional brain atrophy.

The findings suggest that future phase 3 trials should explore the duration of pain relief and headache recurrence, with the goal of developing structured treatment guidelines for PTH.