Neuromuscular

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.

Neurology News Network for the week ending January 6, 2023. [WATCH TIME: 3 minutes]

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

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

The professor of neurology at Cedars-Sinai Medical Center talked about efgartigimod as a novel treatment for chronic inflammatory demyelinating polyneuropathy, with the potential of it being a first-line treatment for patients with the condition. [WATCH TIME: 3 minutes]

The vice president of the Rare Disease Translational Center at the Jackson Laboratory provided commentary on the next steps needed to further understand stathmin-2’s role as a potential disease-modifying biomarker. [WATCH TIME: 3 minutes]

Assessments on PGN-EDODM1's safety, transcript splicing correction, and clinical outcome measures data at the 5 mg/kg dose level from trial is expected this year.

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.

Catch up on any of the neurology news headlines you may have missed over the course of December 2023, compiled all into one place by the NeurologyLive® team.

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.

As part of NeurologyLive®'s Year in Review, we've compiled the most-read feature-length stories that appeared on the website in 2023.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 29, 2023.

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]

As part of NeurologyLive®'s Year in Review, take a look at our most-read news in neuromuscular disorders in 2023.

In 2023, these episodes of the Mind Moments® podcast got the most attention from listeners, with this list brought to you as part of NeurologyLive®'s Year in Review.

As part of NeurologyLive®'s Year in Review, take a look at our most-watched interviews with key opinion leaders in neuromuscular disorders in 2023.

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

Neurology News Network for the week ending December 23, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 22, 2023.

The chief medical officer of Acadia Pharmaceuticals provided commentary on a caregiver analysis assessing beneficial experiences with trofinetide in patients with Rett syndrome. [WATCH TIME: 4 minutes]

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.

Dr Neelam Gupta highlights differences between recently approved complement inhibitors for treatment of myasthenia gravis.

A neuromuscular disorder specialist, Dr Neelam Goyal shares data with efgartigimod, a neonatal Fc receptor inhibitor, for treatment of myasthenia gravis.

The vice president of the Rare Disease Translational Center at the Jackson Laboratory discussed results from a recently published study reinforcing stathmin-2 as a potential therapeutic strategy for ALS. [WATCH TIME: 4 minutes]