American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting (AANEM)

The Meta A. Neumann Professor of Neurology at George Washington University provided an overview of a new phase 3 study testing the efficacy and safety of cladribine versus placebo in generalized myasthenia gravis. [WATCH TIME: 3 minutes]

Pozelimab and cemdisiran work together to block the complement pathway, which plays a key role in gMG, using two different approaches—one suppresses liver production of a key protein, while the other targets it directly with antibodies.

Zilucoplan, approved for generalized myasthenia gravis in 2023, showed consistent complement inhibition, even with additional treatments like IVIg or plasma exchange.

The director of the myasthenia gravis clinic at Yale University provided clinical insight on promising phase 3 data assessing inebilizumab, an FDA-approved therapy for NMOSD, in patients with myasthenia gravis. [WATCH TIME: 4 minutes]

A new analysis of the phase 3 PROPEL study showed that switching to cipaglucosidase alfa/miglustat therapy enhanced outcomes among patients with late-onset Pompe disease.

Using data from observational studies, a recent meta-analysis offered a comprehensive overview of the risk profile of cardiovascular comorbidities in patients with myasthenia gravis.

A new study presented at AANEM 2024 revealed that daily dosing of oral edaravone was well tolerated and did not reveal any new safety concerns in patients with amyotrophic lateral sclerosis.

Long-term use of ravulizumab in patients with generalized myasthenia gravis led to a significant reduction in corticosteroid dependence, highlighting its potential steroid-sparing benefits.

Over an 18-month period, patients showed strengthened scores on Rasch-built Overall Disability Scale, a patient-reported outcome measure of activity and social participation.

The professor and chair of neurology at Virginia Commonwealth University discussed how neurologists are urged to take organized action and provide training on healthcare disparities, with a particular focus on race, ethnicity, and financial barriers to access. [WATCH TIME: 2 minutes]

The professor of neurology at Cedars-Sinai Medical Center talked about efgartigimod as a novel treatment for chronic inflammatory demyelinating polyneuropathy, with the potential of it being a first-line treatment for patients with the condition. [WATCH TIME: 3 minutes]

The director of disability policy at the Muscular Dystrophy Association talked about the future possibility of having an aircraft that accommodates for patients with neuromuscular disorders who use mobility devices. [WATCH TIME: 3 minutes]

The neuromuscular neurologist at the Cleveland Clinic discussed findings from an analysis presented at AANEM 2023 that investigated the safety and efficacy of subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 5 minutes]

Group-level analyses indicated that a combination of cipaglucosidase alfa and miglustat outperformed alglucosidase alfa/placebo in various motor function and patient-reported outcome measures, with notable improvements in walking tests and quality of life aspects like 'ability to move around' and 'energy level.'

The founder of Strategic Advocacy Solutions LLC discussed the critical role of psychosocial support throughout various stages of neuromuscular diseases, emphasizing the impact of transitions on patients' emotional well-being. [WATCH TIME: 5 minutes]

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]

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.

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]

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.

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] 

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.

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 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.

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 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.

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.

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] 

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.

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.

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.