Neuromuscular

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]

Efgartigimod-treated patients consistently outperformed placebo regardless of concomitant medication, thymectomy status, and timing of diagnosis.

After previously showing positive effects on MG-ADL and QMG, ravulizumab continued to show improvements in ocular and respiratory muscle domains in myasthenia gravis.

The director of the Center for Biologics Evaluation Research at the FDA provided an overview of the Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference. [WATCH TIME: 3 minutes]

In a phase 3 study, tofersen failed to meet its primary end point of functional change, but did meet its target engagement of SOD1 protein and reductions in neurofilament light.

More than 90% of patients, ambulatory and non-ambulatory, experienced improvements in Medical Research Council score within 6 months of treatment.

Differences in baseline characteristics and eligible criteria in presymptomatic infants with spinal muscular atrophy led to differential results on motor and nonmotor clinical outcomes.

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 the history of the Muscular Dystrophy Association (MDA).

The FDA has reversed course, deciding to hold an advisory committee meeting ahead of the May 29, 2023, action date for Sarepta’s investigational DMD agent SRP-9001 (also known as delandistrogene moxeparvovec).

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 17, 2023.

Ahead of the 2023 MDA Clinical and Scientific Conference, Anne Connolly, MD, provided perspective on conversations surrounding holistic approaches to neuromuscular diseases.

Jeffery Neul, MD, PhD, director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, shared his reactions and thoughts to the approval of trofinetide.

In honor of Brain Awareness Week, a group of experts in the care of patients with neurological conditions—Ronald C. Petersen, MD, PhD; Brian Grosberg, MD, FAHS; James Beck, PhD; Michael Levy, MD, PhD; Stanley H. Appel, MD —shared their perspectives on hot topics of treatment and management in neurology.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jeffrey L. Neul, MD, PhD. [LISTEN TIME: 12 minutes]

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

The therapy, marketed as Daybue, is the first therapy indicated for Rett syndrome with supporting data from the pivotal phase 3 LAVENDER study.

Neurology News Network for the week ending March 11, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 10, 2023.

Treatment with a lower dose of CNM-Au8 resulted in significantly decreased risk of death, feeding tube placement, assisted ventilation, and all-cause hospitalization, among other survival outcomes.

Batoclimab, a fully human anti-FcRn monoclonal antibody, has demonstrated an ability to significantly alleviate patients’ symptoms and improve quality of life in myasthenia gravis.

Efgartigimod resulted in rapid impact on health-related quality of life, with improvements that were consistent across multiple measures and were similar for the effect seen across 2 treatment cycles.