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.
May 10th 2024
Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 2024.
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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'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 CME: Optimizing Therapy in Parkinson’s Disease
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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
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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 Role of the Complement Proteins in Neurologic Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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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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Advancements and Challenges in Genetic Treatments for Neuromuscular Diseases: John W. Day, MD, PhD
February 12th 2024Ahead of the 2024 MDA Conference, the medical advisor and care center director at Muscular Dystrophy Association talked about the track sessions he will be cochairing at the meeting. [WATCH TIME: 5 minutes]
NeurologyLive® Brain Games: February 11, 2024
February 11th 2024Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is neuromuscular disorders, and specifically, Duchenne muscular dystrophy.
NeuroVoices: Joe Burnett, on FDA Clearance of SmartFrame OR Stereotactic System
February 7th 2024The chief executive officer of ClearPoint Neuro provided clarity on how the recently approved SmartFrame system can enhance the efficiency of operating room procedures, allowing for flexible workflows and image-based corrections.
Transformation of Rett Syndrome Research With the Emergence of Adult Data: Walter E. Kaufmann, MD
February 2nd 2024The adjunct professor of human genetics at Emory University School of Medicine discussed previous research on Rett syndrome that initially provided crucial insight on the neuromuscular condition in adult populations. [WATCH TIME: 2 minutes]
Positive Phase 2 Data Announced for MOMENTUM Study of Duchenne Agent SRP-5051
January 30th 2024Over a 28-week treatment period, the investigational agent demonstrated significantly greater increase in dystrophin expression and exon skipping than previously approved eteplirsen, Sarepta’s first therapy for Duchenne muscular dystrophy.
Easing Neurosurgery With Newly Approved SmartFrame OR Stereotactic System: Joe Burnett
January 28th 2024The chief executive officer of ClearPoint Neuro discussed how the company’s SmartFrame OR Stereotactic System can facilitate neurosurgery and the next steps in integrating it into clinical settings. [WATCH TIME: 4 minutes]
Low-Dose Benzodiazepines Linked With Worsened Disease Progression in Progressive Supranuclear Palsy
January 27th 2024Even after adjusting for indications for benzodiazepine administration and baseline disease severity, results showed greater patient disease worsening while on such medications, regardless of duration and dose.
Caregiving Youth: A Call to Action for Improved Support
January 27th 2024A systematic approach to understanding the needs of the caregiving youth—a growing and less-studied population of caregivers—is needed to better tailor interventions, provide support, and improve care to patients and their families.
A Clinical Preview of 2024 for Neuromuscular Disease: Richard Lewis, MD
January 22nd 2024The professor of neurology at Cedars-Sinai Medical Center shared his thoughts on potential therapies that expand management care options for patients with neuromuscular disease, in particularly for chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 4 minutes]