Neuromuscular

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 primary progressive aphasia.

In recent news, Lexeo Therapeutics' gene therapy candidate LX2006 received FDA fast track designation for patients with Friedreich ataxia cardiomyopathy.

Neurology News Network. for the week ending April 27, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 26, 2024.

A case study of a 52-year-old Korean woman revealed the potential of Nanopore long-read sequencing in diagnosing atypical late-onset ataxia-telangiectasia.

The chief scientific officer at Athira talked about the potential of hepatocyte growth factor compounds in promoting neurotrophic signaling and providing broad neuroprotection across neurodegenerative diseases. [WATCH TIME: 6 minutes]

The chair of neurology at Massachusetts General Hospital talked about results from the phase 2b PARADIGM trial assessing PrimeC, an ALS combination agent that consists of ciprofloxacin and celecoxib. [WATCH TIME: 4 minutes]

After 2 years of treatment, EDG-5506, otherwise known as sevasemten, was well-tolerated, with rapid and sustained decreases in biomarkers of muscle damage.

Expert neurologists share approaches to switching MG treatment due to either poor disease control or adverse events.

Experts in neurology review the varied routes of administration of targeted treatment in MG, highlighting intravenous and subcutaneous formulations and how they can provide more patient-centric care.

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 sleep disorders.

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.