Neuromuscular

The research grant aims to diversify Friedreich ataxia treatments, addressing challenges in trial design and therapeutic strategies for this condition.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 19, 2024.

Expert neurologists discuss the treatment algorithm of MG, focusing on the use of targeted therapies as well as barriers to their use.

Nicholas Silvestri, MD, FAAN, and Tuan Vu, MD, provide an overview of the current FDA-approved targeted therapies for the treatment of MG.

A phase 3b study showed that switching from intravenous C5 inhibitors to subcutaneous zilucoplan in patients with myasthenia gravis was associated with symptom improvement at 12 weeks.

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 on the American Academy of Neurology Annual Meeting.

Patients on riliprubart demonstrated improvements on several measures of disability and impairment, including the INCAT, I-RODS, MRC-SS, and grip strength.

Neurology News Network. for the week ending April 13, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 12, 2024.

All 8 participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change and the Clinician Reported Global Impression of Change scales.

The new double-blind, placebo-controlled study includes approximately 200 patients with ALS who will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells that will be used to manufacture each participant’s NurOwn treatment for the trial.

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles described some of the major resource and policy-level barriers for gene therapy in pediatric neurological conditions. [WATCH TIME: 6 minutes]

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

The first patient enrolled into the phase 1/2a ArthemiR trial assessing ATX-01 myotonic dystrophy type 1 is expected in the second quarter of 2024.

Experts in neurology highlight taking age and quality of life into consideration when approaching treatment of MG, as well as comment on the standard of care for patients.

Expert neurologists discuss the factors that guide treatment selection in MG, shifting the framework to take patient preferences into account.

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles discussed the hesitancy to enroll children with neurodevelopmental issues in early intervention trials without proven products or markers. [WATCH TIME: 5 minutes]

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 on limb girdle muscular dystrophy.

The phase 1 study, dubbed PRO-101, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both patients with ALS and healthy volunteers.

Neurology News Network. for the week ending April 6, 2024. [WATCH TIME: 3 minutes]

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

The executive medical lead at Italfarmaco SpA expressed his confidence in givinostat to mitigate the progression of Duchenne muscular dystrophy, emphasizing its multifactorial mechanism of action. [WATCH TIME: 5 minutes]

Amylyx Pharmaceuticals’ AMX0035, despite its safety profile, did not show significant efficacy among patients with amyotrophic lateral sclerosis in the phase 3 PHOENIX trial.