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.
July 30th 2025
Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.
Evaluating Givinostat's Efficacy and Safety in Duchenne Muscular Dystrophy: Marcello Paglione, PhD
April 3rd 2024The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]
NeuroVoices: Francesco Saccà, MD, PhD, on Crossing Over Dimethyl Fumarate in Friedriech Ataxia
April 3rd 2024The associate professor of neurology at the University of Naples discussed a recently enrolled study assessing the use of dimethyl fumarate, an approved therapy for relapsing MS and psoriasis, in patients with Friedreich ataxia.
Challenges in Identifying Markers for Neurodevelopmental Disorders: Shafali Jeste, MD
April 2nd 2024The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles provided insight on the barriers to prevent neurodevelopmental disorders and the lack of currently validated biomarkers. [WATCH TIME: 4 minutes]
Therapeutic Potential of Dimethyl Fumarate to Treat Friedreich Ataxia: Francesco Saccà, MD, PhD
March 28th 2024The associate professor of neurology at the University of Naples provided context on the mechanism of action for dimethyl fumarate and why there’s belief it can benefit patients with Friedreich ataxia. [WATCH TIME: 4 minutes]
Integrating Genetics and Transitional Support in Muscular Dystrophy Care: Amanda Peltier, MD
March 27th 2024The professor of neurology at Vanderbilt University Medical Center discussed advocating for reinstating personalized resources, expanding genetic testing, and improving transitional care in muscular dystrophy. [WATCH TIME: 5 minutes]
A Patient Perspective on Gene Therapy for Neuromuscular Diseases: Justin Moy
March 26th 2024The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]
Challenges in Transitioning Care for Duchenne Muscular Dystrophy: Diana Castro, MD
March 25th 2024The neuromuscular specialist at the Neurology & Neuromuscular Care Center discussed the pressing need to address the transition of care from pediatric to adult healthcare systems in Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]