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 3rd 2024
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 3, 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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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 Proteins in Neurologic Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological 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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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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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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Gene Editing as a Promising Treatment Approach for Duchenne Muscular Dystrophy: Walter Strapps, PhD
June 30th 2023The chief scientific officer at Liberate Bio discussed how through the advancement of nonviral delivery technologies and nucleic acid therapeutics, genetic medicines can potentially bring relief to patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
Pegcetacoplan to be Discontinued in ALS Following Disappointing Phase 2 Results
June 24th 2023In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.
Benefits and Risks of Opioid Use in Restless Legs Syndrome: John Winkelman, MD, PhD
June 22nd 2023The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital discussed the benefits opioids have for patients with restless legs syndrome, as well as how they can be safely used as part of treatment strategies. [WATCH TIME: 4 minutes]
FDA Approves Subcutaneous Efgartigimod as Treatment for Generalized Myasthenia Gravis
June 20th 2023The basis for the FDA’s decision came from the phase 3 ADAPT-SC study, in which subcutaneous efgartigimod showed a slightly better ability to reduce immunoglobulin compared with its previously approved intravenous formulation.
CNM-Au8 Shows Significant Reductions in Neurofilament in HEALEY ALS Trial
June 15th 2023New data announced by Clene Nanomedicine suggested that the gold nanocrystal therapy reduced plasma neurofilament light chain, which principal investigator Merit Cudkowicz, MD, said, “help advance the design of a phase 3 trial.”
Full Efficacy and Safety Results From Phase 3 LAVENDER Trial of Trofinetide Published
June 12th 2023Results from the phase 3 LAVENDER trial investigating trofinetide (Daybue; Acadia), which supported the first FDA-approved treatment for Rett syndrome, were recently published in Nature Medicine.
Phase 2/3 Study of Blarcamesine in Pediatrics With Rett Syndrome Completes Enrollment
June 8th 2023After discussions with the FDA, the study will assess 2 co-primary end points of change in Rett Syndrome Behavior Questionnaire total score and Clinical Global Impression Improvement Scale score.
Critical Updates to the Treatment of Restless Legs Syndrome: John Winkelman, MD, PhD
June 7th 2023The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital provided insight on new updates to the management of restless legs syndrome, including removing dopamine agonists as first line treatments. [WATCH TIME: 8 minutes]