Marco Meglio

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

The study investigates the potential benefits of ThecaFlex DRx System, an implantable intrathecal catheter, for patients with SMA treated with nusinersen, aiming to reduce the need for serial lumbar punctures and enhance the overall administration experience.

Among a cohort of nearly 500 patients, more than half of caregiver respondents reported reductions in seizure frequency alongside improvements in nonseizure-related outcomes.

The associate director of the Alzheimer’s Disease Research Unit at the Yale School of Medicine provided commentary on positive phase 1 findings assessing ALX-001, a highly selective agent in development for neurodegenerative diseases.

Approved in Europe in late 2022, the 24-hour continuous subcutaneous infusion acts as a novel and effective treatment option for patients with advanced levodopa-responsive Parkinson disease.

In addition to showing no significant dose-response relationship with ischemic stroke risk, milvexian was also associated with an increase in major bleeding events.

Over 144 weeks of treatment, patients on CNM-Au8 demonstrated sustained improvements in low contrast vision, working memory, and information processing speed.

Additional data, including secondary end points and other efficacy assessments, are expected to be presented at an upcoming scientific meeting.

After 6 months of treatment, once every 4-week administration of DYNE-251 reached levels of dystrophin expression, exon skipping, and percent dystrophin positive fibers that exceeded levels reported in a previous trial of eteplirsen, considered the standard of care.

Overall, investigators observed DMPK knockdown, consistent splicing correction, and meaningful function improvement in myotonia while on DYNE-101.

Months after the FDA cleared a phase 2 study in myasthenia gravis, KYV-101 is set to be assessed in a diverse cohort of refractory progressive multiple sclerosis.

Over the 12-week study, treatment with famciclovir failed to reduce the frequency of viral shedding, further adding to the complexity of the role of Epstein-Barr virus in MS.

Over a year period on the combination agent, patients showed significantly amplified Aß42 and Aß40 levels with no significant differences in other biomarkers of ptau, GFAP, or neurofilament light.

Over 76 weeks of treatment, those on 30 mg CNM-Au8 demonstrated significantly reduced plasma neurofilament light coupled with delayed time to morbidity events for the highest at-risk patients.

The senior vice president of medical and clinical affairs for Avadel Pharmaceuticals provided perspective on a recently published analysis demonstrating the benefits of once-nightly sodium oxybate in narcolepsy type 1 and type 2.

Investigators observed no new safety concerns, no cases of Alzheimer-related imaging abnormalities, and no notable changes in laboratory evaluations and EKG.

Steve Hughes, MD, chief medical officer at Avidity Biosciences, provided commentary on recently announced positive topline data for AOC 1044, an investigational agent for Duchenne muscular dystrophy.

Take a look at 5 of the most-anticipated clinical trial data readouts expected in 2024 that neurology health care professionals should keep their eyes on.

Mind Moments®, a podcast from NeurologyLive®, brings you a review of 2023, with insights from Sharon Cohen, MD, FRCPC; Robert A. Hauser, MD, MBA; Alberto Espay, MD, PhD; Carolina Ferreira Atuesta, MD, MSc; and Natalie Goedeker, CPNP. [LISTEN TIME: minutes]

The neurologist at Cleveland Clinic shared thoughts on a recently published study assessing the relationship between blood biomarkers and change in cognitive function and brain volumes in a cohort of professional fighters.

The research aimed to assess FMD prevalence and clinical manifestations in both elderly and younger onset individuals, revealing insights into symptom variations across different age groups.

The newly announced trial, dubbed ReTain, will featured 500 individuals with preclinical Alzheimer disease who will be randomized to ACI-35.030 or placebo for a maximum of 4 years.

The senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical, gave an overview of LILAC-2, a long-term extension study assessing trofinetide (Daybue), the first approved therapy for Rett syndrome.

Elizabeth M. Cassidy, MPH, research project manager at the TSC Alliance, provided clarity on a presentation at AES 2023 examining the association between TSC-associated neuropsychiatric disorders and seizure duration.

In a phase 3 study, patients on omaveloxolone had less physical impairment compared with patients who received placebo, as measured by the modified Friedreich Ataxia Rating Scale.

A-synuclein-positive athletes were similar to α-synuclein-negative athletes on demographics and other serum and blood biomarkers; however, these patients had lower grey matter volumes in the right inferior orbitofrontal, right anterior insula, and right olfactory cortices.

Subgroups of narcolepsy types 1 and 2 had significant reductions in a number of secondary outcomes, including number of sleep stage transitions, number of nocturnal arousals, and improvements in sleep quality.

AOC 1044 was well-tolerated in healthy volunteers, delivering dose-dependent increases in PMO concentrations in skeletal muscle following administration.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jennifer Gudeman, PharmD. [LISTEN TIME: 17 minutes]

The positive data report could potentially build on phase 2 study of Duchenne muscular dystrophy in which CAP-1002 met its primary end point.