Neuromuscular

In new analyses of a pivotal phase 3 study, eplontersen resulted in beneficial effects on neuropathy impairment and quality of life in patients regardless of sex or change in body mass index.

LX2006 shows safety, tolerance, and significant improvements in cardiac biomarkers in FA cardiomyopathy, progressing to higher dose cohorts.

Take a look at some of the most-anticipated clinical trials with expected data readouts in the second half of 2024 that researchers and clinicians in neurology should keep an eye out on.

Emma Ciafaloni, MD, addresses the important role of multidisciplinary care in the treatment of patients with Duchenne muscular dystrophy.

The neurologist and clerkship director at the University of Connecticut provided perspective on the developing role of clerkship directors and the value they provide to neurology departments.

A recent study revealed that pediatric patients with Duchenne and Becker muscular dystrophy have low rates of corticosteroid use.

Additional interim clinical study results comprising 6- and 12-months of treatment data on multiple patients, is expected to be readout in the first quarter of 2025.

Key opinion leaders address the scarcity of randomized controlled trial data for SMA treatments in adult populations, highlighting the availability of real-world data from numerous studies and provide guidance on how to incorporate this information when making treatment decisions for adult patients with spinal muscular atrophy.

Panelists delve into strategies for engaging in conversations with patients about defining and identifying successful therapy outcomes, providing guidance on how to effectively communicate and establish realistic expectations for treatment success in the context of spinal muscular atrophy (SMA).

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network. for the week ending July 20, 2024. [WATCH TIME: 4 minutes]

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

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 multiple system atrophy.

Following the positive data, NeuroSense intends to submit 12-month results to the FDA for regulatory discussions on future path.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 12, 2024.

Patients with CIDP treated with riliprubart reported improved quality-of-life and fatigue measures, as well as reduced neurofilament light levels.

A neurologist discusses how recent approvals of therapies for Duchenne muscular dystrophy have improved patient outcomes.

Emma Ciafaloni, MD, discusses therapies for Duchenne muscular dystrophy that target pathways downstream of dystrophin, focusing on vamorolone and givinostat.

Overall, treated patients showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed volumes.

Panelists share their approaches to counseling adult patients with spinal muscular atrophy (SMA) about treatment expectations.

Key opinion leaders share their strategies for discussing potential changes in therapy and transitioning between treatments with patients, emphasizing the importance of reassuring patients that such changes are intended to provide long-term benefits for managing their spinal muscular atrophy.

The chief executive officer and the chief scientific officer at CureDuchenne talked about a recent webinar that discussed the broad FDA approval of a gene therapy for Duchenne muscular dystrophy, highlighting patient choice and the ongoing efforts to improve treatments. [WATCH TIME: 9 minutes]

Catch up on any of the neurology news headlines you may have missed over the course of June 2024, compiled all into one place by the NeurologyLive® team.

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network. for the week ending July 6, 2024. [WATCH TIME: 4 minutes]