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.
Investigating Losmapimod in Phase 2 Trial for Facioscapulohumeral Muscular Dystrophy
May 15th 2023Leo H. Wang, MD, PhD, FAAN, associate professor of neurology, University of Washington Medical Center, talked about the phase 2 study of losmapimod for facioscapulohumeral muscular dystrophy presented at the 2023 AAN Annual Meeting.
FDA Approves Enzyme Replacement Therapy Pegunigalsidase Alfa for Fabry Disease
May 10th 2023To date, the enzyme replacement therapy had been studied in a comprehensive clinical development program that comprised more than 140 patients with Fabry disease with up to 7.5 years of follow up treatment.
AOC 1001 as a Potential Treatment for Myotonic Dystrophy Type 1
May 9th 2023Nicholas Johnson, MD, MSci, FAAN, division chief of neuromuscular disorders and vice chair of research, Virginia Commonwealth University, provided insight on promising phase 1/2 data of AOC 1001 in myotonic dystrophy type 1.
Evolving Landscape of Pediatric Neurology: Erika Fullwood Augustine, MD, MS
May 5th 2023The associate chief science officer at Kennedy Krieger Institute shared thoughts on the state of pediatric neurology and the significance of being named winner of the Sydney Carter Award. [WATCH TIME: 3 minutes]
AAN Annual Meeting 2023: Top Expert Interviews
May 2nd 2023A group of experts in the care of patients with neurological conditions—Ali Jannati, MD, PhD; Madhav R. Thambisetty, MD, PhD; Daniel Ontaneda, MD, PhD; Peter J. Goadsby, MD, PhD, DSc; Andrew Russman, DO—shared their perspectives on hot topics of treatment and management in neurology from the 2023 American Academy of Neurology Annual Meeting.
Serial Assessments Used in Groundbreaking Brain Study: Imad Najm, MD
May 2nd 2023The director of Cleveland Clinic’s Epilepsy Center at the Cleveland Clinic Neurological Institute provided perspective on the in-depth testing neurologically healthy patients in the Brain Study will undergo. [WATCH TIME: 3 minutes]