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 2nd 2024
Leslie Citrome, MD, MPH, clinical professor of psychiatry and behavioral sciences at New York Medical College, shared his reaction to the recently approved new sprinkle formulation of valbenazine for patients with tardive dyskinesia.
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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FDA Approves Expanded Indication of DaxibotulinumtoxinA to Treat Cervical Dystonia
August 15th 2023In a phase 3 study and open-label extension, patients treated with daxibotulinumtoxinA showed significant improvements in their condition, explained by changes on the Toronto Western Spasmodic Torticollis Rating Scale.
Advancing Care With Exon Skipping Therapies for Duchenne Muscular Dystrophy: Vamshi Rao, MD
August 15th 2023The associate professor of pediatrics in neurology and epilepsy at Northwestern University Feinberg School of Medicine discussed the FDA-cleared phase 1/2 trial of an exon 50 skipping therapy to address the specific mutations causing Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]
Building a Healthy Learning Environment for Young Medical Professionals: Alexandria Reynolds, PhD
August 14th 2023The undergraduate program director at the University of South Carolina discussed the shift in teaching strategies for those in neurology and why more hands-on experience serves students better. [WATCH TIME: 5 minutes]
Dosing Commenced in Phase 3 FORTIFY of Limb-Girdle Muscular Dystrophy Type 2i
August 10th 2023BridgeBio Pharma met with the FDA to discuss the use of glycosylated α-dystroglycan levels as a surrogate end point for its phase 3 trial FORTIFY investigating BBP-418 and believes there is the potential for an accelerated approval.
Subgroup Findings of the NURTURE Study, Importance of CMAP Amplitude in SMA: Thomas Crawford, MD
August 6th 2023The pediatric neurologist at Johns Hopkins Medicine provided commentary on the subgroup findings from the phase 4 NURTURE study of nusinersen (Spinraza; Biogen) in presymptomatic spinal muscular atrophy. [WATCH TIME: 3 minutes]
Second Patient Dosing Recommended for TSHA-102 Gene Therapy Trial in Rett Syndrome
August 2nd 2023Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.
Ropinirole Demonstrates Safety and Efficacy in ALS Progression in 6-Month Trial
August 2nd 2023Investigators concluded that the growing and testing of motor neurons from patient-derived induced pluripotent stem cells could be clinically used for the prediction of ropinirole’s efficacy as a treatment for patients with ALS.
NeuroVoices: Thomas Crawford, MD, on Continued Benefit of Early Initiated Nusinersen in SMA
August 2nd 2023The pediatric neurologist at Johns Hopkins Medicine provided clarity on the recently published 5-year update of the NURTURE study, and the importance of treating spinal muscular atrophy at its presymptomatic stages.