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.
Check out some of NeurologyLive®'s featured content in the carousel below.
September 25th 2023
In an integrated meta-analysis of the HEALEY ALS platform trial and RESCUE-ALS trial, 30mg of CNM-Au8 resulted in 59% decreased risk of mortality among participants with ALS compared with PRO-ACT matched placebo over long-term follow-up.
Recognizing Rett Syndrome Early to Improve Long-Term Management Outcomes
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Shaping the Management of Multiple Sclerosis – The Potential for BTK Inhibitors in Clinical Practice
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Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
10/13/2023
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From Clinical Trials to Clinical Practice – Incorporating Screening and Assessment Strategies for the Early Diagnosis of Alzheimer Disease
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Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making
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Addressing Healthcare Inequities in Stroke Care: How Can We Play an Active Role in Improving Outcomes for All Patients?
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2022 4th Annual International Congress on the Future of Neurology
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Understanding Best Care Practices for Hemorrhagic Stroke – A Focus on the Role of DOAC Reversals
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Collaborating Across the Continuum™ : Managing Relapsing MS – A Focus on the Utility of CD20-Targeted B-Cell Therapy
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Patient, Provider, and Caregiver Connection™: Challenges in Diagnosis and Management for Patients with ADHD During the COVID-19 Pandemic
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Expert Illustrations & Commentaries™: A Family Physicians’ Guide to Migraine Treatment – Emerging Therapies and Evolving Paradigms
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PER® Postgame™: MS Meeting Updates – Integrating the Latest Data to Optimize Care in the COVID-19 Era
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Medical Crossfire®: What Strategies Can the Care Team Implement to Optimize the Management of Pediatric Patients with NF1?
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Patient, Provider, & Caregiver Connection: Individualizing Care in Multiple Sclerosis – Understanding Patient Challenges and the Role of Innovative Treatment
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BURST CME™: Taking ALS Management to the Next Level
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FAQs in MS: Exploring Provocative Questions From MS Meeting Updates
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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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Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care
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NeuroVoices: Crystal Proud, MD, on Improving SMA Outcomes Through a Combination Approach
July 12th 2023The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided perspective on the phase 4 RESPOND study assessing nusinersen in individuals with SMA who already received gene therapy.
Revolutionizing Care for Myasthenia Gravis With the Shift to Subcutaneous Therapy
July 4th 2023Vera Bril, MD, FRCPC, lead investigator of the MycarinG study, shared her reactions to recent approval of rozanolixizumab-noli, the first approved therapy for both subtypes of generalized myasthenia gravis in adults.
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]
Biohaven Submits New Drug Application for Troriluzole as Spinocerebellar Ataxia Type 3 Therapy
June 25th 2023In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.
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.