Neuromuscular

Enrollment in the dose level 2 expansion cohort is expected to complete early in Q3 2024, with initial strength and functional data anticipated in the second half of 2024.

In this episode, the panel discusses the emerging challenges for neurology departments, the shortage of neurologists, and the ways to disseminate between useful and useless information amid a busy career.

John England, MD, and Lisa Merlin, MD, commented on the significant changes to neurology over the years and how education aligns with the latest in treatments advancements and understanding of neurological disorders.

To ensure participant safety, all boys who received the gene therapy in the clinical program will be followed up for long-term safety monitoring.

The duo provided insight on a number of key sessions clinicians and neurology educators should attend at the upcoming AUPN Annual Meeting.

Panelists John England, MD, FAAN, and Lisa Merlin, MD, discuss the main objectives of the upcoming AUPN Annual Meeting, set to take place September 14th in Orlando, Florida.

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

Neurology News Network. for the week ending July 27, 2024. [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 on congenital muscular dystrophy.

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

Over a 48-week period, patients treated with low molecular weight dextran sulfate compound had minimal changes in ALS Functional Rating Scale and ALS Assessment Questionnaire.

The expanded indication, supported by a phase 2 open-label trial, includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or asymptomatic.

Pending positive results from an early-stage trial, a mid-stage study evaluating MyoPAXon’s effect on other targeted muscles and muscle function will be initiated.

Treated patients demonstrated improvements on the Bayley-4 in cognition, communication, and motor function that exceeded those seen in natural history studies of Angelman syndrome.

Across all studies, including 2 of the largest recorded for CIDP, patients unanimously demonstrated a preference for SCIG over IVIG.

Medical professionals explore the challenges and considerations associated with patients with spinal muscular atrophy (SMA) transitioning to independent living, emphasizing the importance of maintaining continuity of care and addressing potential adherence issues to ensure optimal disease management.

Panelists examine the available real-world data (RWD) on the use of risdiplam in adult patients with spinal muscular atrophy (SMA) from various studies conducted worldwide, discussing its potential efficacy and safety as a treatment option for adult SMA patients.

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

Amifampridine enhances neuromuscular transmission and relieves muscle weakness, showing promise for Lambert-Eaton myasthenic syndrome and other neuromuscular junction disorders.

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 progressive multiple sclerosis.

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

In new analyses of a pivotal phase 3 study, eplontersen resulted in beneficial effects on neuropathy impairment and quality of life in patients regardless of sex or change in body mass index.

LX2006 shows safety, tolerance, and significant improvements in cardiac biomarkers in FA cardiomyopathy, progressing to higher dose cohorts.

Take a look at some of the most-anticipated clinical trials with expected data readouts in the second half of 2024 that researchers and clinicians in neurology should keep an eye out on.