Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 26, 2023.

Fifty-four percent of patients with ALS carried at least one detrimental common variant or repeat expansion, highlighting the clinical impact of gene variants as modifiers in ALS.

Robust, sustained reductions in key pharmacodynamic biomarker were observed; however, this did not lead to functional benefits on any of the scales used in the trial.

Submitted under the accelerated approval pathway, if approved, SRP-9001 would become the first marketed gene therapy specific for Duchenne muscular dystrophy.

The consultant and founder of Accelerating NeuroVentures talked about the pridopidine arm from the HEALEY ALS Platform trial, which showed significant benefits in speech measures. [WATCH TIME: 5 minutes]

In honor of ALS Awareness Month, held May 2023, get caught up on some of the latest news in amyotrophic lateral sclerosis as the NeurologyLive® team shares some of our data updates.

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

The chief executive officer of Cognixion talked about the Cognixion ONE Axon, a wireless, wearable brain-computer interface that combines EEG technology with an augmented reality headset for patients with neurodegenerative diseases. [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 sleep and sleep disorders.

The phase 1/2 MARINA trial assessed the activity of AOC 1001 across key biomarkers, including spliceopathy, an important biomarker for myotonic dystrophy type 1, and knockdown of DMPK mRNA.

Neurology News Network for the week ending May 20, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 19, 2023.

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

Neurological rehabilitation, or neurorehabilitation, can be described as the use of rehabilitation interventions to improve or maintain function and quality of life in the context of neurological conditions.

Comprised of the recharge-free Proclaim SCS family and the rechargeable Eterna SCS platform, the approval for Abbott’s spinal cord stimulation system was backed by the large-scale, DISTINCT study.

Following thymectomy, more than 80% of patients with myasthenia gravis showed symptom improvement, as demonstrated by changes in Myasthenia Gravis Foundation of America scores.

At the 2023 AAN Annual Meeting, the neuromuscular disease specialist and professor of neurology at the University of Kansas Medical Center talked about the phase 3 study investigating ataluren for Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Leo H. Wang, MD, PhD, FAAN, associate professor of neurology, University of Washington Medical Center, talked about the phase 2 study of losmapimod for facioscapulohumeral muscular dystrophy presented at the 2023 AAN Annual Meeting.

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 headache and migraine.

The FDA is set to make a final decision later this month and, if approved, SRP-9001 would be the first gene therapy specific to treat Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 12, 2023.

To date, the enzyme replacement therapy had been studied in a comprehensive clinical development program that comprised more than 140 patients with Fabry disease with up to 7.5 years of follow up treatment.

Nicholas Johnson, MD, MSci, FAAN, division chief of neuromuscular disorders and vice chair of research, Virginia Commonwealth University, provided insight on promising phase 1/2 data of AOC 1001 in myotonic dystrophy type 1.

Melanie Leitner, PhD, consultant and founder of Accelerating NeuroVentures, talked about the findings from regimen D of the HEALY ALS platform trial assessing pridopidine.