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 1st 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 1, 2025.
Dosing Underway for PIERRE Pivotal Study of ThecaFlex DRx System and Nusinersen
January 11th 2024The study investigates the potential benefits of ThecaFlex DRx System, an implantable intrathecal catheter, for patients with SMA treated with nusinersen, aiming to reduce the need for serial lumbar punctures and enhance the overall administration experience.
Neurologist’s Role in Addressing Healthcare Disparities: A. Gordon Smith, MD, FAAN
January 7th 2024The professor and chair of neurology at Virginia Commonwealth University discussed how neurologists are urged to take organized action and provide training on healthcare disparities, with a particular focus on race, ethnicity, and financial barriers to access. [WATCH TIME: 2 minutes]
Dyne Reports Positive Phase 1/2 Data for Duchenne Agent DYNE-251
January 6th 2024After 6 months of treatment, once every 4-week administration of DYNE-251 reached levels of dystrophin expression, exon skipping, and percent dystrophin positive fibers that exceeded levels reported in a previous trial of eteplirsen, considered the standard of care.
Steps to Validate Stathmin-2 as an ALS-Specific Biomarker: Cathleen Lutz, PhD
January 4th 2024The vice president of the Rare Disease Translational Center at the Jackson Laboratory provided commentary on the next steps needed to further understand stathmin-2’s role as a potential disease-modifying biomarker. [WATCH TIME: 3 minutes]
Clinical Potential of AOC 1044 in Treating Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping
January 2nd 2024Steve Hughes, MD, chief medical officer at Avidity Biosciences, provided commentary on recently announced positive topline data for AOC 1044, an investigational agent for Duchenne muscular dystrophy.