The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
September 1st 2025
Here's some of what is coming soon to NeurologyLive® this week.
Long-Term IV Edaravone Not Associated With Disease-Modifying Benefit in ALS
January 13th 2022Despite not providing additional, clinically relevant benefit for patients with amyotrophic lateral sclerosis as an add-on to standard therapy, the intravenous treatment was feasible and well tolerated.
Spasticity, Dystonia Commonly Under-Identified in Children With High Cerebral Palsy Risk
January 13th 2022Only 30% of the observed cohort of more than 150 children had their hypertonia type specified as spasticity and/or dystonia by age 5 years, highlighting a need for additional education efforts.
The Effect of Efgartigimod’s Approval on the Myasthenia Gravis Landscape: James F. Howard, MD
December 29th 2021The Distinguished Professor of Neuromuscular Disease at University of North Carolina School of Medicine offered his insight into the newly approved Argenx agent, marketed as Vyvgart. [WATCH TIME: 3 minutes]
GM1 Treatment PBGM91 Shows Positive Interim Data in Phase 1/2, Additional Cohorts Planned
December 22nd 2021Two patients with late infantile GM1 gangliosidosis in Cohort 1 received a low dose of the treatment, reporting no serious adverse events. Passage Bio noted it plans to move forward with 2 additional cohorts.