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 16th 2024
Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-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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Assessing Phase 2/3 Trial Safety and Efficacy of AMO-02 for Myotonic Dystrophy: Mike Snape, PhD
October 6th 2023The chief scientific officer of AMO Pharma discussed results from a recent clinical trial evaluating the safety and efficacy impact of AMO-02 on children and adolescents with congenital myotonic dystrophy. [WATCH TIME: 6 minutes]
Clinical Viewpoint on Newly Approved Combination Therapy for Pompe Disease: Barry J. Byrne, MD, PhD
October 5th 2023The chief medical advisor at the Muscular Dystrophy Association gave his reaction to Amicus Pharmaceuticals’ recently FDA approved 2-part therapy for patients living with Pompe disease. [WATCH TIME: 7 minutes]
SARA Scale Scores Strongly Predictive of Friedreich Ataxia Progression
October 1st 2023Over a 4-year follow-up, SARA progression was substantially linear. Age at evaluation also influenced the speed of SARA progression, while disease duration did not improve the prediction of the statistical model.
Neuro News Roundup: LGMD Awareness Day – Expert Insight and Latest Literature
September 30th 2023In honor of Limb-Girdle Muscular Dystrophy Awareness Day, held September 30, 2023, get caught up on some of the latest news in LGMD, with data updates and expert insights all in one place from the NeurologyLive® team.
Mechanistic Approach Behind AMX0035 in Progressive Supranuclear Palsy: Gunter Hoglinger, MD
September 29th 2023The professor of neurology and translational neuroscientist at Ludwig-Maximillian’s University Munich detailed the logic behind the ORION study, a new trial assessing AMX0035, an approved therapy for ALS, in patients with progressive supranuclear palsy. [WATCH TIME: 4 minutes]
FDA Approves Cipaglucosidase Alfa and Miglustat as First Two-Component Therapy for Pompe Disease
September 29th 2023In a phase 3 setting, the 2-component therapy showed positive trends or clinically meaningful improvements on motor and respiratory functions compared with previously approved enzyme replacement therapy.
NeurologyLive® Clinician of the Month Spotlight: Peter B. Kang, MD, FAAN, FAAP
September 28th 2023As part of our monthly clinician spotlight, NeurologyLive® highlighted neueomuscular expert Peter Kang, MD, director of the Paul and Sheila Wellstone Muscular Dystrophy Center and professor of neurology at the University of Minnesota.
New Interim Data Reported on DNL310 for Hunter Syndrome Treatment in Phase 1/2 Trial
September 26th 2023Additional biomarker findings from up to 2 years of DNL310 treatment suggest positive changes in adaptive behavior and cognition as well as improvement in auditory function among pediatric patients with Hunter syndrome.
CNM-Au8 Shows Significant Long-Term Survival Improvement in HEALEY ALS Platform Trial
September 25th 2023In 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.