Neuromuscular

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.

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

In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.

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 neuromuscular disorders.

In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.

Neurology News Network for the week ending June 24, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 23, 2023.

Marketed as Elevidys, the gene therapy is approved for ambulatory pediatric patients aged 4 to 5 with Duchenne based on expression of micro-dystrophin.

The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital discussed the benefits opioids have for patients with restless legs syndrome, as well as how they can be safely used as part of treatment strategies. [WATCH TIME: 4 minutes]

Biomarker data at 49 weeks continued to demonstrate that DNL310 enabled rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers.

The basis for the FDA’s decision came from the phase 3 ADAPT-SC study, in which subcutaneous efgartigimod showed a slightly better ability to reduce immunoglobulin compared with its previously approved intravenous formulation.

In one of the first phase 3 trials of primary mitochondrial myopathy, elamipretide failed to meet its primary end points; however, a subgroup of patients with nuclear DNA defects saw improvement in 6-minute walk test, raising further questions.