Latest Conference Coverage

Evaluating Positive Phase 1 Trial Data for Narcolepsy Agent ALKS 2680

Understanding Zilucoplan’s Positive Impact on Fatigue in Myasthenia Gravis: Michael D. Weiss, MD, FAAN

The director of the neuromuscular diseases division at the University of Washington discussed findings from a new post hoc analysis presented at AANEM 2023 that showed that zilucoplan treatment significantly reduced fatigue in patients with myasthenia gravis. [WATCH TIME: 6 minutes]

Targeting Neurotrophic Factors and Preclinical Results of ALS Agent ATH-11055: Kevin Church, PhD

The 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]

Sub-Analysis of CHAMPION MG Study Reveals No Differences in Ravulizumab Treatment Based on Prior Immunoglobulin Use

Recent findings from an analysis of the CHAMPION MG study suggest that ravulizumab continues to be an effective therapy for patients with generalized myasthenia gravis, regardless of whether they had received prior IVIg treatment.

Overviewing Preliminary Data of ABvac40, a Vaccine for Alzheimer Disease: Jose Terencio, PhD

The vice president of Innovation and New Technologies at Grifols provided perspective on the interim phase 2 findings of ABvac40, a vaccine in development for patients with Alzheimer disease. [WATCH TIME: 3 minutes]

NeuroVoices: Joseph Foss, MD, on NTRX-07 and CBR2-Targeting Therapies for Alzheimer Disease

The chief medical officer of NeuroTherapia provided insight on promising phase 1b data assessing NTRX-07, a CBR2-targeting agent in development for patients with early-stage Alzheimer disease.

Neurologists' Perspectives on Healthcare Disparities in Myasthenia Gravis: A. Gordon Smith, MD, FAAN

The 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

Recent 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

The 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

Among 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.

Early Zilucoplan Treatment Maintains Long-Term Benefit in Generalized Myasthenia Gravis

In a new post hoc analysis of RAISE-XT, early responders to zilucoplan maintained their response throughout the long-term open-label extension, irrespective of baseline characteristics.

Zilucoplan Shows Reduction in Fatigue in the Phase 3 Trials for Generalized Myasthenia Gravis

Zilucoplan demonstrated significant and sustained improvements in myasthenic fatigue when compared with the placebo in the phase 3 RAISE study and its extension trial, RAISE-XT.

Addressing the Invisible Symptoms of Duchenne Muscular Dystrophy: Mathula Thangarajh, MD, PhD

The assistant professor at Virginia Commonwealth University provided comment on her presentation from AANEM 2023, and the need to raise awareness towards nonneuromuscular symptoms of Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]

The Payor Dilemma With Accessing Effective Therapies for NMOSD: Bruce Cree, MD, PhD, MAS, FAAN

The clinical research director of the UCSF Multiple Sclerosis Center talked about challenges in accessibility for the 3 approved therapies for NMOSD caused by third-party payor resistance as well as the importance of educating clinicians about the effectiveness of inebilizumab. [WATCH TIME: 3 minutes]

Efgartigimod Shows Consistent Safety Profile Across Immunoglobulin G-Mediated Autoimmune Diseases in Trials

The safety and tolerability of efgartigimod was consistent across different indications and doses, showing comparable rates of treatment-related adverse events with placebo in clinical trials for myasthenia gravis, primary immune thrombocytopenia, and pemphigus.

Zilucoplan Shows Reduction in MG Worsening in Phase 3 RAISE Study for Generalized Myasthenia Gravis

A 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.

Results on Efgartigimod for Chronic Inflammatory Demyelinating Polyneuropathy Treatment: Richard Lewis, MD

The professor of neurology at Cedars-Sinai Medical Center talked about findings from the ADHERE trial assessing efgartigimod, a human IgG1 antibody Fc fragment, as a treatment for patients with chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 6 minutes] 

Social Determinants of Health Lead to Greater Suboptimal Treatment Response in Myasthenia Gravis

A 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

In 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.

Creatine Kinase Levels Not Considered Risk Factor for COVID-19 Post-Acute Sequelae

Elevated creatine kinase levels were not found to be a prognostic factor for the development of PASC in encephalopathic COVID-19 patients, according to a retrospective chart review analysis.

Treatment Inequality Issues Identified for Patients With Generalized Myasthenia Gravis

In 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

The 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]

Decreased Neurofilament Light in ATTR Polyneuropathy Observed With Vutrisiran and Patisiran

Vutrisiran and patisiran treatment led to noticeable reductions in neurofilament light, indicating a potential biomarker of treatment response in hATTR amyloidosis patients.

Subcutaneous Efgartigimod PH20 Demonstrates Efficacy for Generalized Myasthenia Gravis in Open Label ADAPT-SC+ Trial

Presented at the 2023 AANEM meeting, new findings suggest that subcutaneous efgartigimod could be a valuable treatment option for patients with myasthenia gravis, with well-tolerated cycles and improved daily living activities.

Open-Label PROPEL Study Results Highlight Longterm Impact of Cipaglucosidase Alfa and Miglustat in Late-Onset Pompe Disease

The phase 3 PROPEL study demonstrated the long-term effectiveness of cipaglucosidase alfa/miglustat in late-onset Pompe disease, with improved 6-minute walk distance and lung function.

The Potential of Nipocalimab as a Treatment for Autoantibody Mediated Diseases: Hong Sun, MD, PhD

The senior director and global compound development team leader at Janssen discussed research on nipocalimab, an investigational agent for patients with myasthenia gravis that's shown promising data to this point. [WATCH TIME: 2 minutes]

Latozinemab Shows No Impact on Disease Progression in C9orf72 Frontotemporal Dementia Despite increasing Progranulin Expression

latozinemab treatment for C9orf72-associated frontotemporal dementia showed no significant impact on disease progression, although the treatment was generally safe and well-tolerated.

The Role of Biomarkers in Myasthenia Gravis Diagnosis and Treatment: Hong Sun, MD, PhD

The senior director and global compound development team leader at Janssen talked about the importance of biomarkers in detecting an earlier diagnosis of myasthenia gravis and the significant unmet medical need for better treatment options. [WATCH TIME: 3 minutes]

NeuroVoices: David Greeley, MD, on AR1001, an Oral PDE5 Inhibitor for Alzheimer Disease

David Greeley, MD, chief medical officer at AriBio and study author, provided commentary on the mechanism of action of AR1001, its promising effect on ptau181, and the next steps in its development.

Designing More Effective Proof-of-Concept Studies for Progressive MS Therapies: Robert J. Fox, MD

The vice-chair for research at Cleveland Clinic’s Neurological Institute discussed the way therapies are currently assessed for progressive MS and the need for disease-specific biomarkers. [WATCH TIME: 3 minutes]