Neuromuscular

In recognition of International Pompe Day, held on April 15, 2023, get caught up on some of the latest news in Pompe disease as the NeurologyLive® team shares some of our data updates.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 31, 2023.

The director of the center for gene therapy at Nationwide Children's Hospital talked about the challenges and opportunities of gene therapies for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

Similar to data reported at 35 weeks, patients on eplontersen continued to see consistent reductions in serum transthyretin concentration for up to 66 weeks of treatment.

The senior statistical scientist and director of consulting at Berry Consultants talked about innovative clinical trial design in ALS research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, the professor of neurology at the University of Minnesota talked about the current understanding and challenges of Limb Girdle muscular dystrophy from a clinical perspective. [WATCH TIME: 5 minutes]

A. Gordon Smith, MD, FAAN, recounts the challenges of treating patients with myasthenia gravis. He also discusses care disparities and the need for rapid disease modification.

A. Gordon Smith, MD, FAAN, reviews prevalence and subtypes of myasthenia gravis. He also discusses how different antibodies contribute to the pathophysiology of the disease.

The senior vice president of research at The ALS Association talked about the complexities of ALS and the importance of collaboration in research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Time to milestones such as invasive and noninvasive ventilation, speech-generating devices, and hospice care took significantly longer while on intravenous edaravone.

The research associate professor in the department of physical therapy at the University of Florida talked about physical therapy inventions for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

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

NurOwn, an autologous mesenchymal stromal cell approach for ALS, failed to meet its primary end point in a pivotal phase 3 trial, but showed promising results on less severe forms of the disease in subsequent analyses.

The child neurologist at the University of Colorado School of Medicine talked about her presentation on end of life care for patients with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 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 the history of the Muscular Dystrophy Association (MDA).

As a recap from MDA 2023, get caught up on some of the latest news in neuromuscular diseases as the NeurologyLive® team shares some of our data updates.

Neurology News Network for the week ending March 25, 2023. [WATCH TIME: 3 minutes]

At the 2023 MDA Conference, a neurology nurse practitioner in the Neuromuscular Division at Washington University in St. Louis talked about adverse event management of gene therapies in Duchenne muscular dystrophy. [WATCH TIME: 2 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 24, 2023.

At the 2023 MDA Conference, the executive vice president and chief research officer of MDA talked about a new program from the MDA to tackle ultra-rare neuromuscular diseases. [WATCH TIME: 5 minutes]

Durability of treatment response on reachable workspace was observed among those who continued losmapimod throughout the 96-week period and those who switched from placebo.

The associate professor of pediatrics at the University of Colorado School of Medicine talked at the 2023 MDA Conference about how digestive health impacts patients with Duchene Muscular Dystrophy. [WATCH TIME: 4 minutes]

In participants who received placebo during the parent studies, rapid improvements were observed as early as week 1 after switching to zilucoplan, and continued through week 12 of the extension period.

The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center discussed the types of resources clinicians can pull from to expand neuropalliative care for patients with neuromuscular disorders. [WATCH TIME: 3 minutes]

The 12-person Dephi panel reviewed data from 3 clinical trials of delandistrogene maxeparvovec (SRP-9001; Sarepta), and developed considerations for the management of vomiting, myocarditis, acute liver injury, and immune-mediated myositis.

The committee all agreed that tofersen presents a favorable risk-benefit profile in this particularly challenging patient population.

After results indicated significant slowing of ALS disease progression, Coya noted it plans to file an IND with the FDA in the second half of 2023 for a potential clinical study thereafter.

The professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.

The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center provided perspective on current state of end-of-life care for neuromuscular disorders and areas to improve upon. [WATCH TIME: 4 minutes]

The chief medical officer at ML Bio Solutions spoke about the advances in neuromuscular diseases such as limb girdle muscular dystrophy at the 2023 MDA Conference. [WATCH TIME: 3 minutes]