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.
April 16th 2024
A phase 3b study showed that switching from intravenous C5 inhibitors to subcutaneous zilucoplan in patients with myasthenia gravis was associated with symptom improvement at 12 weeks.
The Expanding Role of Fluid Biomarkers in the Diagnosis and Management of Patients With Alzheimer Disease
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Expert Illustrations & Commentaries™: Targeting Immune Cells to Treat Multiple Sclerosis
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Improving Care for Patients with Motor Complications of Parkinson Disease: Role of Technology and Data
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Clinical Consultations™: Considerations for Customizing Care Plans for Patients with Parkinson Disease Psychosis
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Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
September 15, 2024
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Mastering MS: Translating Evidence into Optimal Management Plans
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Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care
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Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes
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5th Annual International Congress on the Future of Neurology®
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Clinical Consultations™: Managing Depressive Episodes in Patients with Bipolar Disorder Type II
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Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
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Medical Crossfire®: Understanding the Advances in Bipolar Disease Treatment—A Comprehensive Look at Treatment Selection Strategies
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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
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Burst CME: Optimizing Therapy in Parkinson’s Disease
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'REEL’ Time Patient Counseling: The Diagnostic and Treatment Journey for Patients With Bipolar Disorder Type II – From Primary to Specialty Care
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
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Addressing Healthcare Inequities: Bridging the Gap in Multiple Sclerosis – A Focus on Clinical and Healthcare Disparities in Black Patients
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Individualizing Treatment for Patients with Generalized Myasthenia Gravis
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Patient, Provider & Caregiver Connection™: Reducing the Burden of Parkinson Disease Psychosis with Personalized Management Plans
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Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
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AMX0035 Improves Pancreatic Function and Glycemic Control in Wolfram Syndrome, Phase 2 Results Show
April 11th 2024All 8 participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change and the Clinician Reported Global Impression of Change scales.
FDA Agrees to New Specialized Protocol for Phase 3b Study of ALS Agent NurOwn
April 10th 2024The new double-blind, placebo-controlled study includes approximately 200 patients with ALS who will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells that will be used to manufacture each participant’s NurOwn treatment for the trial.
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]
Enhancing Multidisciplinary Care for Neuromuscular Disorders: Amanda Peltier, MD
March 25th 2024The professor of neurology at Vanderbilt University Medical Center talked about the importance of multidisciplinary care centers to improve accessibility and quality of care for patients with neuromuscular disorders. [WATCH TIME: 5 minutes]