Neuromuscular

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

A. Gordon Smith, MD, FAAN, discusses how to assess for treatment success and why the patient perspective is so important in deciding whether to change therapies.

A. Gordon Smith, MD, FAAN, talks about how new agents and emerging data are enabling clinicians to personalize care for patients with myasthenia gravis.

CSL Behring's subcutaneous immune globulin is the first and only immune globulin available in prefilled syringes for patients with primary immunodeficiency or chronic inflammatory demyelinating polyneuropathy.

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

Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.

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.

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

The chief medical officer of QurAlis talked about the potential of QRL-201 in patients with ALS from her talk presented at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 5 minutes]

In patients with high C-reactive protein levels treated with NP001, investigators observed a 64% slower rate of decline in forced vital capacity.

A. Gordon Smith, MD, FAAN, discusses the findings and implications of phase 3 ADAPT trial and open-label extension study evaluating the use of efgartigimod in the treatment of patients with myasthenia gravis.

A. Gordon Smith, MD, FAAN, explores the rationale for targeting the neonatal Fc receptor and the mechanisms for efgartigimod for use in the treatment of myasthenia gravis.

The multicenter, multiple-ascending dose trial will feature 64 patients with ALS who will be assessed on safety and pharmacokinetic outcomes.

A. Gordon Smith, MD, FAAN, considers how rituximab and eculizumab fit into the current standard of care for myasthenia gravis.

A. Gordon Smith, MD, FAAN, discusses the role of acetylcholine esterase inhibitors, immunosuppressive agents, IV immunoglobulin, corticosteroids, as well as newer agents in the treatment of myasthenia gravis.

The neuromuscular physician at Virginia Commonwealth University talked about the promising future of gene therapy and access to resources in Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.

In the phase 2 study, patients with Guillian-Barré syndrome will receive imlifidase on day 1, followed with intravenous immunoglobulin on days 3-7, with outcomes of safety and change in disability.

At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]

The co-founder and chief executive officer of MyoGene Bio talked about the advantages of gene editing over traditional gene therapy as a long term treatment for neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 3 minutes]

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

The research physical therapist at Nationwide Children’s Hospital talked about the role of physical therapy in the changing landscape of care 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 multiple sclerosis (MS).

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

The associate professor of neurology at Columbia University spoke at the 2023 MDA conference about the unmet needs of patients with ALS and the potential of new gene therapies. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Robin Ryther, MD, PhD, director of the Rett Spectrum Clinic at Washington University in St. Louis School of Medicine, shared her response to the recently approved therapy trofinetide for Rett syndrome.

The associate professor of neurology at the University of Texas Health Science Center at Houston talked about advances in the classification of inflammatory myopathies at the recently concluded 2023 MDA conference. [WATCH TIME: 4 minutes]

A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.