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.
August 1st 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 1, 2025.
Targeting Neurotrophic Factors and Preclinical Results of ALS Agent ATH-11055: Kevin Church, PhD
November 8th 2023The chief scientific officer at Athira Pharma talked about new preclinical results presented at the 2023 NEALS Annual Meeting on the investigational therapy ATH-1105 for patients with amyotrophic lateral sclerosis. [WATCH TIME: 5 minutes]
Inclusive ALS Grant Expands Access to CNM-Au8 Therapy for ALS: Karen S. Ho, PhD
November 8th 2023The vice president of translational medicine at Cene Nanomedicine talked about a recently funded grant that is focused on inclusivity and the collection of real-world data on the efficacy of CNM-Au8 in various stages of amyotrophic lateral sclerosis. [WATCH TIME: 7 minutes]
Neurologists' Perspectives on Healthcare Disparities in Myasthenia Gravis: A. Gordon Smith, MD, FAAN
November 7th 2023The professor and chair of neurology at Virginia Commonwealth University discussed a study conducted among neurologists that revealed healthcare disparities in the treatment of myasthenia gravis, particularly related to access to care and therapeutics. [WATCH TIME: 5 minutes]
Study Reveals Racial Disparities in Acute Care Utilization Among Patients With Myasthenia Gravis
November 7th 2023Recent research, presented at the 2023 AANEM Annual Meeting, emphasized the potential need for patient support programs targeting at-risk populations to enhance disease management and reduce acute care utilization in myasthenia gravis.
How Clinicians can Advocate for Patients Beyond the Clinic: Michael Lewis, MA
November 7th 2023The director of disability policy at the Muscular Dystrophy Association discussed his lecture at AANEM 2023 on how clinicians can advocate for their patients to help improve accessibility and health equity. [WATCH TIME: 7 minutes]
Majority of Patients With Generalized Myasthenia Gravis Fail to Meet Clinical Trial Criteria
November 7th 2023Among 67 patients with generalized myasthenia gravis studied from January 2021 to June 2023, 89.5% failed to meet clinical trial inclusion criteria, with the most common reason being a Myasthenia Gravis Activities of Daily Living score of less than 5.
Zilucoplan Shows Reduction in MG Worsening in Phase 3 RAISE Study for Generalized Myasthenia Gravis
November 3rd 2023A post hoc analysis of the phase 3 RAISE study evaluating zilucoplan, a complement C5 inhibitor, showed significantly fewer patients experienced worsening and fewer requiring rescue therapy while on medication compared with placebo.
Social Determinants of Health Lead to Greater Suboptimal Treatment Response in Myasthenia Gravis
November 2nd 2023A retrospective study presented at the 2023 AANEM meeting revealed that social determinants of health are linked to poorer treatment outcomes in myasthenia gravis patients, emphasizing the need for identifying and supporting at-risk individuals.
Vutrisiran Shows Benefit Across Baseline Severities in Phase 3 HELIOS-A Study
November 2nd 2023In a recent post hoc analysis presented at the AANEM meeting, findings highlight the positive impact of vutrisiran on quality of life, disability, gait speed, and nutritional status across different neuropathy impairment score quartiles.
Treatment Inequality Issues Identified for Patients With Generalized Myasthenia Gravis
November 2nd 2023In a recent online survey of 150 participating neurologists in the US, 74.7% reported that patients with generalized myasthenia gravis found it difficult to afford their prescribed therapies, and 60.0% noted an increased likelihood of exacerbation or hospitalization for these patients.
Understanding the ADAPT-SC Results of Subcutaneous Efgartigimod: Tuan Vu, MD
November 2nd 2023The division director of Neuromuscular Medicine & EMG at USF Health provided an overview of the open-label findings from ADAPT-SC, a phase 3 study assessing subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 3 minutes]