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.
Leading Efforts in Advancing Neurodevelopmental Disorder Research: Walter E. Kaufmann, MD
September 20th 2023The adjunct professor of human genetics at Emory University School of Medicine talked about efforts from various communities in neurological disorders, such as Rett syndrome, to develop new measures for research. [WATCH TIME: 3 minutes]
Efforts Made to Improve the Rett Syndrome Behavior Questionnaire
September 11th 2023The Rett Syndrome Behavior Questionnaire’s implementation as global outcome measure in FDA-approved trials was the result of its comprehensive nature since it includes behavior-related symptom questions as well as questions for regular behaviors.
Restoring Dystrophin Protein Production in DMD With Exon Skipping AOC Therapy: Steve Hughes, MD
September 5th 2023The chief medical officer at Avidity Biosciences talked about how its exon skipping AOC therapies are designed to address the lack of functional dystrophin protein among patients with Duchenne muscular dystrophy to improve muscle function. [WATCH TIME: 4 minutes]
ALS Agent CNM-Au8 Displays Survival Benefit and Delay of Clinical Worsening in 2 Year Follow-Up
September 2nd 2023Findings from an open-label extension trial among 45 patients with ALS showed a significant median survival benefit of 19.3 months and a significant 52% decreased risk of ALS clinical worsening events after treatment.
Creating Efficient, Well-Rounded Neurology Departments
August 31st 2023A panel of former AUPN presidents detailed how advances in neurology have changed the way the clinical specialty is taught, and why collaboration among leadership remains vital to creating an effective neurology department. [WATCH TIME: 7 minutes]
Duchenne Agent Pamrevlumab Fails to Meet Primary End Point in Phase 3 LELANTOS-2 Trial
August 31st 2023Despite pamrevlumab being generally safe and well tolerated, the potential first-in-class connective tissue growth factor inhibitor antibody did not meet its primary endpoint in a phase 3 trial for ambulator Duchenne muscular dystrophy.