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.
August 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
Acute Flaccid Myelitis: Drawing Attention Amid the Peak Months
September 6th 2022Olwen C. Murphy, MBBCh, and Matthew R. Vogt, MD, PhD, discussed the current clinical understanding of AFM and the need to recognize this condition amid the “peak” months of infection: August, September, and October.
Changes in Practice, Perspective on Developmental Milestones for Children: Paul Lipkin, MD
September 3rd 2022The director of medical outpatient services at the Kennedy Krieger Institute provided insight on how clinicians have typically assessed children who fail to meet milestones, and how that might change with new guidelines. [WATCH TIME: 3 minutes]
Vamorolone Shows Efficacy in Duchenne Muscular Dystrophy, Safety Advantages Over Prednisone
September 3rd 2022Compared with placebo, vamorolone-treated patients showed significantly better outcomes on several secondary and exploratory end points, such as the NorthStar Ambulatory Assessment and time to climb 4 stairs.
Behavioral Impairments More Commonly Found in SOD1 ALS Than Cognitive Impairments
September 2nd 2022Using Strong et al’s classification criteria, the percentage of SOD1+ and SOD1- patients with altered cognitive and/or behavioral profiles were similar; however, cognitive and behavioral impairment differentiated between groups.
Overview of New CDC Developmental Milestones for Young Children: Paul Lipkin, MD
August 30th 2022The director of medical outpatient services at the Kennedy Krieger Institute provided insight on the recently announced revisions to the “Learn the Signs. Act Early.” CDC developmental milestones for children. [WATCH TIME: 4 minutes]
Overcoming the Complexities With Gene Therapy in ALS: Anna Underhill, BS
August 18th 2022The postdoctoral researcher at King’s College London provided background on why gene therapies have not seen as much success in treating ALS, and the need for additional validation of genetic mutations. [WATCH TIME: 3 minutes]
BLA Application to Be Submitted for ALS Agent NurOwn Following Phase 3 Statistical Corrections
August 18th 2022The correction in Muscle & Nerve resulted in a statistically significant treatment difference of more than 2 points for average change from baseline in ALSFRS-R in the prespecified efficacy subgroup of with a baseline score of at least 35.
Differences in the Genetic Mutations of ALS: Anna Underhill, BS
August 17th 2022The postdoctoral researcher at King’s College London provided insight on the immunologic differences between genes linked with ALS, and the important aspects to consider for gene-targeted therapies. [WATCH TIME: 3 minutes]