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.
November 27th 2023
Here's some of what is coming soon to NeurologyLive® this week.
Collaborating Across the Continuum™ : Managing Relapsing MS – A Focus on the Utility of CD20-Targeted B-Cell Therapy
View More
Patient, Provider, and Caregiver Connection™: Challenges in Diagnosis and Management for Patients with ADHD During the COVID-19 Pandemic
View More
Expert Illustrations & Commentaries™: A Family Physicians’ Guide to Migraine Treatment – Emerging Therapies and Evolving Paradigms
View More
PER® Postgame™: MS Meeting Updates – Integrating the Latest Data to Optimize Care in the COVID-19 Era
View More
Medical Crossfire®: What Strategies Can the Care Team Implement to Optimize the Management of Pediatric Patients with NF1?
View More
Patient, Provider, & Caregiver Connection: Individualizing Care in Multiple Sclerosis – Understanding Patient Challenges and the Role of Innovative Treatment
View More
FAQs in MS: Exploring Provocative Questions From MS Meeting Updates
View More
BURST CME™: Taking ALS Management to the Next Level
View More
The Expanding Role of Fluid Biomarkers in the Diagnosis and Management of Patients With Alzheimer Disease
View More
Expert Illustrations & Commentaries™: Targeting Immune Cells to Treat Multiple Sclerosis
View More
Improving Care for Patients with Motor Complications of Parkinson Disease: Role of Technology and Data
View More
Clinical Consultations™: Considerations for Customizing Care Plans for Patients with Parkinson Disease Psychosis
View More
Mastering MS: Translating Evidence into Optimal Management Plans
View More
Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care
View More
Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes
View More
Creating Efficient, Well-Rounded Neurology Departments
August 31st 2023A panel of former AUPN presidents detailed how advances in neurology have changed the way the clinical specialty is taught, and why collaboration among leadership remains vital to creating an effective neurology department. [WATCH TIME: 7 minutes]
Duchenne Agent Pamrevlumab Fails to Meet Primary End Point in Phase 3 LELANTOS-2 Trial
August 31st 2023Despite pamrevlumab being generally safe and well tolerated, the potential first-in-class connective tissue growth factor inhibitor antibody did not meet its primary endpoint in a phase 3 trial for ambulator Duchenne muscular dystrophy.
Nusinersen Exhibits Long-Term Safety and Efficacy Across Wide Spectrum of SMA Severity
August 31st 2023Findings from a real-world study among 120 patients with SMA suggests that nusinersen administration is safe and well tolerated, with only 30% of the patients mostly experiencing mild adverse events.
EXPLORE44's Approach to Rare Disease Treatment for Duchenne Muscular Dystrophy: Steve Hughes, MD
August 30th 2023The chief medical officer at Avidity Biosciences discussed the phase 1/2 EXPLORE44 trial, assessing an investigational treatment for Duchenne muscular dystrophy, and highlighted the potential of the treatment based on the recent FDA orphan drug and fast track designations. [WATCH TIME: 4 minutes]
NeurologyLive® Clinician of the Month Spotlight: Gary Hisch, MD
August 28th 2023As part of our monthly clinician spotlight, NeurologyLive® highlighted expert Gary Hisch, MD, pediatric neurologist at Cleveland Clinic, who oversees programs for neuromuscular disorders such as for patients with spinal muscular atrophy.
Genome Sequencing Shows Strong Efficacy in Diagnosing Infant Genetic Disorders
August 22nd 2023In a clinical study diagnosing genetic disorders, genomic sequencing did not report 19 variants found by a targeted neonatal gene-sequencing test and the targeted panel did not report 164 variants recognized by genomic sequencing.
Exon 44 Skipping Agent AOC 1044 Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy
August 16th 2023AOC 1044, an exon 44-targeting agent, holds potential to fill a major unmet need for patients with Duchenne muscular dystrophy, as there are currently there are no approved therapies approved targeting this mutation.
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]