Neuromuscular

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 the European Committe for Treatment and Research in Multiple Sclerosis.

In preclinical data, treatment with PGN-EDODM reduced pathogenic nuclear foci by 54% per nuclei and liberated muscleblind like splicing regulator 1, resulting in a greater than 68% correction of downstream transcript missplicing events.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 13, 2023.

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

The director of the Duke ALS Clinic at Duke Health discussed the advances in drug development for ALS and ways to evolve future trials to get optimal therapeutic benefit. [WATCH TIME: 3 minutes]

In a phase 3 study, patisiran met its primary and first secondary end point, demonstrating significant benefit in functional capacity, quality of life, and health status, in comparison with placebo.

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

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 sleep disorders.

After 1 year of treatment with risdiplam, 81% of the 26-infant cohort were able to sit independently for 30 seconds, a milestone not typically reached in SMA.

Neurology News Network for the week ending October 7, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 6, 2023.

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well-tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.

The chief scientific officer of AMO Pharma discussed results from a recent clinical trial evaluating the safety and efficacy impact of AMO-02 on children and adolescents with congenital myotonic dystrophy. [WATCH TIME: 6 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Gunter Hoglinger, MD. [LISTEN TIME: 14 minutes]

The phase 3 study is expected to include 86 children with ataxia-telangiectasia who will be assessed on changes in a rescored modified International Cooperative Ataxia Rating Scale.

The chief medical advisor at the Muscular Dystrophy Association gave his reaction to Amicus Pharmaceuticals’ recently FDA approved 2-part therapy for patients living with Pompe disease. [WATCH TIME: 7 minutes]

After being diagnosed with acute flaccid myelitis more than 10 years ago, patient advocate Sarah Todd Hammer spoke on the ways in which disability should be viewed and discussed.

In a cross-study analysis, omaveloxolone slowed progression in FA by at least 50% each year for 3 years compared with corresponding data from an external control group in a natural history study.

Previous data on the 25 mg cohort of CTI-1601 showed significant reductions in frataxin levels among treated individuals with Friedreich ataxia.

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

Over a 4-year follow-up, SARA progression was substantially linear. Age at evaluation also influenced the speed of SARA progression, while disease duration did not improve the prediction of the statistical model.

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 epilepsy and seizure disorders.

Years of genetic research has uncovered more about the underpinnings of limb-girdle muscular dystrophy, leading to potential disease-modifying therapies in the near future.

In honor of Limb-Girdle Muscular Dystrophy Awareness Day, held September 30, 2023, get caught up on some of the latest news in LGMD, with data updates and expert insights all in one place from the NeurologyLive® team.

Neurology News Network for the week ending September 30, 2023. [WATCH TIME: 4 minutes]