Marco Meglio

Despite conditional approval in Europe since 2014, ataluren's authorization has faced rejections and is still investigational in the US.

Approved in 2021 under the accelerated approval pathway, aducanumab’s path has been met with controversy and lack of industry support.

VCA-894A, a novel antisense oligonucleotide, exhibits a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2.

Despite initial enthusiasm, novel treatments for Duchenne muscular dystrophy face discontinuation challenges and lack long-term clinical efficacy data.

Over a 28-week treatment period, the investigational agent demonstrated significantly greater increase in dystrophin expression and exon skipping than previously approved eteplirsen, Sarepta’s first therapy for Duchenne muscular dystrophy.

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 movement disorders.

Even after adjusting for indications for benzodiazepine administration and baseline disease severity, results showed greater patient disease worsening while on such medications, regardless of duration and dose.

The proposed NSD-ISS staging system for Parkinson's disease, focusing on neuronal α-synuclein pathology, aims to enhance trial design and drug development.

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Orrin Devinsky, MD. [LISTEN TIME: 18 minutes]

In total, 82% of patients were interested in more convenient treatments, emphasizing the importance of shared decision-making and open discussions with physicians in rare disease management.

Although there were no significant differences in the primary end secondary end points, patients with mRS scores of 0 to 2 at day 90 seemed to show better outcomes in the cell therapy group, particularly for those with ischemic core volumes of 50 mL or greater.

The chief executive officer of Aruna Bio provided context on a newly accepted phase 1/2 trial assessing the company’s neural-derived exosome agent AB126 in patients with acute ischemic stroke.

Overall, the results revealed that light therapy helped to alleviate sleep efficiency, sleep quality, depressed mood, caregiver burden, and agitated behavior.

Noninvasive brain stimulation techniques, particularly transcranial direct current stimulation (tDCS), prove safe and effective for cerebellar ataxia, enhancing motor coordination.

Using the validated Migraine-Related Stigma questionnaire, investigators observed significant between-group differences in disability, interictal burden, and quality of life based on the amount of migraine-related stigma experienced.

Among the disease-modifying drugs (DMDs) for multiple sclerosis (MS), glatiramer acetate was associated with a lower hazard of hypertension, presenting a potential positive impact on cardiovascular health.

The study provided the first class IIb evidence of the long-term benefits of deep brain stimulation in patients with Parkinson disease, emphasized by improved quality of life and reduced medication requirements.

In a phase 2 proof-of-concept study, IHL-42X, a drug for obstructive sleep apnea, more significantly reduced apnea-hypopnea index and oxygen desaturation index than placebo.

Data supported a positive causal association between narcolepsy and schizophrenia and a reverse causal relationship between narcolepsy and major depressive disorder.

In this multicenter, randomized, controlled trial, corticosteroids were found to be superior to clobazam in improving cognition in children with epileptic encephalopathy with spike-wave activation in sleep.

The strong association observed at APOE in this analysis was consistent with observations from other amyloid-lowering therapies, which also found an elevated incidence of ARIA among APOE ε4 carriers.

PARADIGM, an ongoing phase 2 placebo-controlled trial, will have additional biomarker data released in the first half of 2024.

The director of NYU Langone’s Comprehensive Epilepsy Center provided perspective on recently published data evaluating the potential mechanisms of sudden deaths in toddlers.

Using a sample of 350 pregnancies, the maintenance of natalizumab during pregnancy past the 30th week had both positive and negative impacts, suggesting a risk-benefit discussion may be needed before patients begin treatment.

The pediatric REVEAL trial assesses THSA-102 in 2 cohorts of pediatric patients with Rett syndrome due to MECP2 loss-of-function mutation, with results expected in mid-2024.

Neurology News Network for the week ending January 13, 2024. [WATCH TIME: 4 minutes]

Good functional outcome at 90 days was more prevalent among patients on argatroban plus antiplatelet therapy vs antiplatelet alone.

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Cathleen Lutz, PhD. [LISTEN TIME: 18 minutes]

Brexpiprazole was generally well tolerated over 12 weeks in this vulnerable patient population, with documented efficacy that supported its approval in early 2023.

Given the low rates of adverse events and benefits to overall health, modifiable risk-reduction strategies could have a significant impact on public health.