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.
Check out some of NeurologyLive®'s featured content in the carousel below.
AT-GAA’s Conceivable Benefits for Pompe Disease and the Need for More Options
September 9th 2022Barry J. Byrne, MD, PhD, the associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida; and the chief medical advisor to the Muscular Dystrophy Association, offered his perspective on the upcoming FDA decision on AT-GAA (Amicus Pharma).
FDA Lifts Hold on Phase 2 Trial of SRP-5051 in DMD Amenable to Exon 51 Skipping
September 6th 2022The hold was originally placed on part B of the phase 2 MOMENTUM trial in Duchenne muscular dystrophy following a June 2022 report of hypomagnesemia after treatment with high-dose SRP-5051. Going forward, Sarepta will adjust its global trial protocol.
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]