Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 21, 2023.

Based on the positive findings, the company intends to submit a new drug application to the FDA before the end of 2023, with regulatory filings in additional markets to follow in 2024.

The 52-week, dose-ascending trial will have data read out in the first half of 2024, with long-term safety and efficacy evaluated over a 5-year period following completion of the initial trial.

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 Alzheimer's Association.

After 4 years of treatment, more than 95% of infants on risdiplam maintained the ability to swallow, an aspect of everyday life that is commonly lost within 2 years in patients with SMA.

Neurology News Network for the week ending July 15, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 14, 2023.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided background on findings from the phase 4 RESPOND study, and the potential of combining therapeutic approaches in SMA. [WATCH TIME: 4 minutes]

The highest proportions of patients with severe ocular impairment at baseline showed greater improvements in symptom severity on ravulizumab in comparison with placebo after 26 weeks.

Nearly half of patients treated with zilucoplan were considered responders on Myasthenia Gravis Activities of Daily Living (MG-ADL) at the first week, suggesting a rapid onset of efficacy.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided perspective on the phase 4 RESPOND study assessing nusinersen in individuals with SMA who already received gene therapy.

Patients with generalized myasthenia gravis treated with efgartigimod consistently exceeded treatment compared with placebo regardless of gender in age, disease duration, BMI, and thymectomy.

Accounting for baseline disease covariates, NurOwn-treated participants had reduced neurofilament light values from baseline to week 20 compared with placebo (P <.05).

Higher levels of the n-3 fatty acid eicosapentaenoic acid and the n-6 fatty acid linoleic acid were associated with a lower risk of death during follow-up.

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 stroke and cerebrovascular disease.

Building on positive 1-year data, 24-month data showed statistically significant differences in the Performance of the Upper Limb scale in treated patients in the open-label extension relative to those on placebo in the double-blind period.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 7, 2023.

On average, younger and older participants who were unable to sit without support improved from baseline on CHOP-INTEND scores over a 183-day period.

Patients on Hyqvia experienced longer time to relapse compared with those on placebo, with Kaplan-Meier estimate curves separating early, at approximately week 4.

The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital provided perspective on the current major research priorities for the treatment of restless legs syndrome. [WATCH TIME: 3 minutes]

Recent advancements have the potential to significantly improve the lives of individuals with myasthenia gravis and contribute to the broader understanding of autoimmune diseases

Vera Bril, MD, FRCPC, lead investigator of the MycarinG study, shared her reactions to recent approval of rozanolixizumab-noli, the first approved therapy for both subtypes of generalized myasthenia gravis in adults.

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 multiple sclerosis.

Neurology News Network for the week ending July 1, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 30, 2023.

The chief scientific officer at Liberate Bio discussed how through the advancement of nonviral delivery technologies and nucleic acid therapeutics, genetic medicines can potentially bring relief to patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

In a phase 3 study, rozanolixizumab demonstrated statistically significant differences vs placebo on primary and secondary outcomes over a 43-day treatment period.