News

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.

If approved, ABBV-951 will become the first subcutaneous delivery of carbidopa/levodopa, often considered the standard of care for symptom management for patients with Parkinson disease.

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.

The vice president of Health Economic and Outcomes Research at Acadia Pharmaceuticals discussed the ongoing challenges with lowering mortality in Parkinson disease, and the need to improve caregiver burden. [WATCH TIME: 4 minutes]

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.

Anne Marie Morse, DO, a pediatric neurologist and sleep medicine specialist at Geisinger Medical Center, talked about the challenges that adolescents with narcolepsy face in their social relationships.

The neurologist and migraine expert from NYU Langone Health shared her reactions to the recently approved zavegepant nasal spray (Zavzpret; Pfizer) for the treatment of migraine. [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).

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).

Mechanistically, reboxetine and a combination of reboxetine and oxybutynin improved pharyngeal collapsibility and respiratory control.

Patients with cerebellar ataxia made riskier decisions than controls, suggesting this may be a unique cerebellar cognitive symptom.

Neurology News Network for the week ending March 18, 2023. [WATCH TIME: 4 minutes]

In honor of World Sleep Day, held March 17, 2023, get caught up on some of the latest news in sleep disorders 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 17, 2023.

The director of the Visual Outcomes Laboratory at Cedars Sinai talked about the current treatments used in MS and the risks associated with them at 2023 ACTRIMS Forum. [WATCH TIME: 3 minutes]

Here's the latest multidisciplinary MS research published online first in the International Journal of MS Care.

The neurologist at Mayo Clinic, in Jacksonville, Florida, spoke about inebilizumab as a treatment for NMOSD and other autoimmune diseases, such as encephalitis, in the ExTINGUISH trial. [WATCH TIME: 3 minutes]