Neuromuscular

Over a 32-week treatment period, patients on trofinetide continued to show improvements on the primary efficacy outcomes of Rett Syndrome Behavior Questionnaire and Clinical Global Impression-Improvement total scores.

Although not powered for statistical significance, results showed improvements in ALS Functional Rating Scale and slow vital capacity after treatment with PrimeC.

The vice president of the Rare Disease Translational Center at the Jackson Laboratory provided context on a recently published study suggesting restoration of stathmin-2 as a therapeutic approach for ALS.

Dr Neelam Goyal describes types of myasthenia gravis, its presentation, and differential diagnosis of myasthenia gravis.

A neuromuscular disorder specialist, Dr Neelam Goyal discusses prevalence and pathophysiology of myasthenia gravis (MG).

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 the history of the American Epilepsy Society.

Neurology News Network for the week ending December 1, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 1, 2023.

Givinostat, a HDAC inhibitor, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial.

Despite additional information submitted, the FDA continues to impose a clinical hold on Entrada Therapeutics' ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy.

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 general neurology.

Neurology News Network for the week ending November 25, 2023. [WATCH TIME: 4 minutes

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 24, 2023.

Pixyl.Neuro, a CE-marked medical device, is designed to be integrated into the radiology workflow, providing clinicians with diagnostic support of neuroinflammatory and neurodegenerative disorders.

The FDA has scheduled an in-person meeting for December 6th to discuss the regulatory path for BrainStorm Cell Therapeutics’ NurOwn as a potential treatment for patients with amyotrophic lateral sclerosis.

Ahead of the 2024 MDA Conference, the vice president of public policy and advocacy at the Muscular Dystrophy Association talked about conference advocacy collaboration, access to treatments, and the state of healthcare. [WATCH TIME: 6 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 November 17, 2023.

The director of disability policy at the Muscular Dystrophy Association talked about the future possibility of having an aircraft that accommodates for patients with neuromuscular disorders who use mobility devices. [WATCH TIME: 3 minutes]

The neuromuscular neurologist at the Cleveland Clinic discussed findings from an analysis presented at AANEM 2023 that investigated the safety and efficacy of subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 5 minutes]

Group-level analyses indicated that a combination of cipaglucosidase alfa and miglustat outperformed alglucosidase alfa/placebo in various motor function and patient-reported outcome measures, with notable improvements in walking tests and quality of life aspects like 'ability to move around' and 'energy level.'

In addition to myasthenia gravis, KYV-101 is being explored in other disorders like systemic sclerosis, multiple sclerosis, and lupus nephritis.

Long-term nicotinamide riboside use in patients with ataxia telangiectasia showed safety, improved motor coordination, and eye movements, suggesting potential disease attenuation.

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

The founder of Strategic Advocacy Solutions LLC discussed the critical role of psychosocial support throughout various stages of neuromuscular diseases, emphasizing the impact of transitions on patients' emotional well-being. [WATCH TIME: 5 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 Parkinson disease.

Steve Hughes, MD, chief medical officer at Avidity, provided perspective on recently announced positive phase 1/2 and OLE data evaluating AOC 1001, an investigational antisense oligonucleotide, in patients with myotonic dystrophy type 1.

Neurology News Network for the week ending November 11, 2023. [WATCH TIME: 3 minutes]