Neuromuscular

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.

At the 2023 MDA conference, the professor for human genetics and neurology at the University of Miami Miller School of Medicine talked about the role of genetics in neuromuscular diseases and potential therapies. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, the scientific research portfolio director at Muscular Dystrophy Association detailed a session on collaborative research in ALS and ongoing efforts in the field. [WATCH TIME: 3 minutes]

Namita A. Goyal, MD, a principal investigator on the pivotal phase 3 trial of NurOwn, talked about the importance of the upcoming FDA AdComm meeting and its impact on the ALS community.

The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]

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

The director of clinical outcomes and research development in neurology at Stanford University spoke about the paradigm shift in neuromuscular therapy interventions 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 Parkinson disease (PD).

Over a 24-week treatment period, reldesemtiv failed to show a significant effect vs placebo on the primary end point of ALS Functional Scale-Revised, as well as other key secondary end points.

Neurology News Network for the week ending April 1, 2023. [WATCH TIME: 4 minutes]

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]