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 18th 2024
ION541, an antisense oligonucleotide, failed to demonstrate impact on outcomes of function, breathing, and strength, in addition to no impact on neurofilament light.
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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Unraveling the Complexities of Cerebral Palsy and Building Hope for a Brighter Future
March 24th 2022During Cerebral Palsy Awareness Month, leaders in the field expressed their love for the CP community, the progress made in managing the disorders, and the steps needed to advance care into a realm never before seen.
Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD
March 23rd 2022The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]
Subcutaneous Efgartigimod Shows Noninferiority to IV Formulation in Generalized Myasthenia Gravis
March 23rd 2022Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).
Improving and Expanding Access to ALS Treatment Options: Calaneet Balas, MSc, MBA
March 23rd 2022The president and chief executive officer of The ALS Association provided commentary on the most pertinent issues with managing patients with ALS and the ways to improve current treatment standards. [WATCH TIME: 4 minutes]
The Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD
March 22nd 2022The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]
BBP-418 Demonstrates Significant Impact on Root Cause of Limb-Girdle Muscular Dystrophy Type 2i
March 22nd 2022Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.
Successes and Challenges in the New Era of Genetic Medicine: Nicholas E. Johnson, MD, MSci
March 20th 2022The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University spoke to the ongoing success in the development of gene-mediated therapies and the challenges that come along with treating rare neuromuscular disease. [WATCH TIME: 3 minutes]
NeurologyLive® Brain Games: March 20, 2022
March 20th 2022Test 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 optic neuritis and neuromyelitis optica spectrum disorder (NMOSD).
Zolgensma Reinforces Efficacy in SPR1NT Results of Patients With SMA and 3 Copies of SMN2
March 19th 2022The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.
Understanding the Newest Therapeutic Advances in Neuromuscular Diseases
March 17th 2022Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.
MDA 2022 Highlights the Therapeutic Advances for Neuromuscular Diseases: Donald S. Wood, PhD
March 17th 2022The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]
Managing New Therapies for Neuromuscular Diseases: Jinsy Andrews, MD, MSc
March 16th 2022The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]