Neuromuscular

In a recent online survey of 150 participating neurologists in the US, 74.7% reported that patients with generalized myasthenia gravis found it difficult to afford their prescribed therapies, and 60.0% noted an increased likelihood of exacerbation or hospitalization for these patients.

The division director of Neuromuscular Medicine & EMG at USF Health provided an overview of the open-label findings from ADAPT-SC, a phase 3 study assessing subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 3 minutes]

Vutrisiran and patisiran treatment led to noticeable reductions in neurofilament light, indicating a potential biomarker of treatment response in hATTR amyloidosis patients.

Presented at the 2023 AANEM meeting, new findings suggest that subcutaneous efgartigimod could be a valuable treatment option for patients with myasthenia gravis, with well-tolerated cycles and improved daily living activities.

The phase 3 PROPEL study demonstrated the long-term effectiveness of cipaglucosidase alfa/miglustat in late-onset Pompe disease, with improved 6-minute walk distance and lung function.

Recent research has suggested a role for thyroid hormones and their receptor, thyrotropin, in muscle regeneration and development, making a case for further investigation in DMD.

Over a 52-week treatment period, SRP-9001 demonstrated robust evidence for a clinically meaningful benefit across several prespecified functional secondary end points in comparison with placebo.

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

Neurology News Network for the week ending October 28, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 27, 2023.

With the FDA approval, vamorolone (Agamree) becomes the first-in-class dissociative steroid therapy for patients with Duchenne muscular dystrophy. Catalyst Pharmaceuticals plans to launch the treatment in the first quarter of 2024.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with James F. Howard, MD. [LISTEN TIME: 16 minutes]

Diego Torres-Russotto, MD, chair of neurology at Baptist Health Miami Neuroscience Institute, provided an overview of the upcoming 11th Annual Miami Neuroscience Symposium, a 3-day educational event held in early November.

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 rare and genetic neurological disorders.

Neurology News Network for the week ending October 21, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 20, 2023.

The chief of the Movement Disorders Division at Mass General Hospital discussed the challenges medical students face in pathing their career and the importance of a strong mentor figure. [WATCH TIME: 6 minutes]

With the approval, zilucoplan becomes the first once-daily subcutaneous C5 complement inhibitor available for adults with generalized myasthenia gravis.

The pediatric neuromuscular neurologist at Arkansas Children's Hospital and associate professor of pediatrics at the University of Arkansas for Medical Sciences talked about findings from a phase 1/2 study on dosing 3 pediatric patients with Duchenne muscular dystrophy with REGENXBIO’s RGX-202. [WATCH TIME: 4 minutes]

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

AOC 1001 consistently demonstrated directional improvements across multiple aspects of myotonic dystrophy type 1, including measures of myotonia, strength, function, and patient reported outcome.

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.