Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 4, 2023.

NS-050/NCNP-03, an exon skipping investigational therapy, has been cleared by the FDA for the initiation of a phase 1/2 clinical study for the treatment of Duchenne muscular dystrophy.

The pediatric neurologist at Johns Hopkins Medicine discussed the importance of early diagnosis in SMA, and the notable barriers that come with obtaining disease-modifying therapies. [WATCH TIME: 4 minutes]

Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.

Investigators concluded that the growing and testing of motor neurons from patient-derived induced pluripotent stem cells could be clinically used for the prediction of ropinirole’s efficacy as a treatment for patients with ALS.

The pediatric neurologist at Johns Hopkins Medicine provided clarity on the recently published 5-year update of the NURTURE study, and the importance of treating spinal muscular atrophy at its presymptomatic stages.

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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 epilepsy and seizure disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 28, 2023.

At 19 weeks, eplontersen-treated patients showed substantial and sustained reductions in serum TTR concentration compared with baseline.

Caution is warranted for older patients with Duchenne muscular dystrophy who are receiving gene therapy, according to details of a patient death in a single patient trial.

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Neurology News Network for the week ending July 22, 2023. [WATCH TIME: 3 minutes]

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.

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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.