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

Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at Wake Forest School of Medicine, discussed a new, innovative trial assessing the therapeutic potential of self-administered subcutaneous rozanolixizumab as a treatment for myasthenia gravis.

These results highlight the age range and ambulation status for which viltolarsen is a key treatment option for DMD patients amenable to exon 53 skipping.

The study, presented at the 2024 AANEM) Annual Meeting, evaluated 52 patients who underwent surgery for carpal tunnel syndrome and explored the comparative effectiveness of the Conway and Zeidman scales in predicting patient outcomes.

The multicenter, 24-week study will feature 20 patients with MG, testing changes on MG-ADL as well as several other secondary outcomes, including patient-reported assessments.

Overall, subcutaneous efgartigimod was safe and effective in patients with seropositive MG, demonstrated by consistent improvements in Myasthenia Gravis-Activites of Daily Living.

For more than a 2-year treatment period, 95% of patients reported taking at least 95% of their daily medications with zilucoplan.

The professor in residence at UCLA Health discussed the critical considerations when deciding appropriate patients for gene therapies, specifically AAV vectors, in treating muscular dystrophies. [WATCH TIME: 4 minutes]

The professor in residence at UCLA Health provided clinical insight on the limitations of certain gene therapy vehicles for muscular dystrophies, and the need for better vectors that are more myotropic. [WATCH TIME: 3 minutes]

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on the advantages of a flipped classroom, and why neurology educators should consider this approach. [WATCH TIME: 2 minutes]

Judith Thompson, PharmD, MPH, CPHQ, rare disease population health strategy lead at UCB, discussed the association between social determinants of health and delayed diagnosis/misdiagnosis in myasthenia gravis.

The director of the myasthenia gravis clinic at Yale University discussed the therapeutic potential of inebilizumab, an FDA-approved treatment for NMOSD, in myasthenia gravis, based on data from the phase 3 MINT study.

The director of the Muscular Dystrophy Clinic at UCSF Benioff Children’s Hospital provided insight on promising data from the phase 2/3 VIBRANCE-MG study assessing investigational nipocalimab in adolesents with myasthenia gravis. [WATCH TIME: 3 minutes]

Amy Tsou, MD, MSc, program director at the National Institutes of Neurological Disorders and Stroke, discussed the increasing cost of prescription drugs in the US and its impact on healthcare providers, including those who treat patients with neurologic conditions.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lawrence Robinson, MD. [LISTEN TIME: 15 minutes]

The rare disease population health strategy lead at UCB provided clinical insight on some of the unique challenges of diagnosing myasthenia gravis, and some of the early signs clinicians and non-specialists should look out for. [WATCH TIME: 3 minutes]

In a small sample population of adolescents with myasthenia gravis, nipocalimab met its primary end point, showing a significant reduction in total serum immunoglobuin over a 24-week period.

The director of the myasthenia gravis clinic at Yale University provided additional insight on the MINT study of inebilizumab in myasthenia gravis, some of the subanalyses within, and next plans in the drug’s development. [WATCH TIME: 5 minutes]

The senior scientist at Sunnybrook Research Institute talked about both the opportunities and challenges with the shift of technological advancement in medical education, especially in fostering critical thinking and managing the vast influx of information. [WATCH TIME: 4 minutes]

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on his lecture given at AANEM 2024, focusing on the challenges and opportunities of teaching the next generation of practitioners.

Constantine Farmakidis, MD, an associate professor of neurology at the University of Kansas Medical Center, provided clinical commentary on a subanalysis of a phase 3 study assessing nipocalimab, an investigational agent, in generalized myasthenia gravis.

Study investigator Henry Kaminski, MD, provided clinical insight on a unique trial assessing the efficacy and safety of oral cladribine tablets, an FDA-approved medication for multiple sclerosis, in patients with myasthenia gravis.

A recent analysis of the phase 3 ADHERE trial demonstrated the clinical benefit of subcutaneous efgartigimod PH20 in patients with chronic inflammatory demyelinating polyneuropathy.

Tuan Vu, MD, a professor of neurology at the University of South Florida, provided brief commentary on topline data of the phase 3 VIVACITY-MG3 study presented at AANEM 2024.

The phase 3 study is expected to include 110 patients with seronegative myasthenia gravis who will be randomly assigned to IV efgartigimod or placebo for a 5-week follow-up, followed by an open-label extension.

The assistant professor of neurology at Wake Forest School of Medicine provided clinical insight on MG0020, a phase 3 trial assessing a self-administration of rozanolixizumab through a syringe driver. [WATCH TIME: 3 minu]

Despite balanced baseline characteristics, patients ranged from low to high number of rozanolixizumab treatment cycles, suggesting the need for a personalized approach.

Findings showed that both fixed cycles and every-other-week dosing regimens of efgartigimod were well tolerated and effective in improving clinical outcomes in patients with generalized myasthenia gravis.

The PREVAIL trial, presented at the 2024 AANEM meeting, is investigating gefurulimab, a bispecific nanoantibody designed to inhibit complement activation, as a potential treatment for patients with generalized myasthenia gravis.

The neuromuscular specialist at the University of Tennessee Medical Center provided clinical insight on a retrospective analysis studying the long-term corticosteroid patterns of approved treatments for generalized myasthenia gravis. [WATCH TIME: 5 minutes]

Rozanolixizumab demonstrated efficacy in patients with generalized myasthenia gravis aged at least 65 years, despite a higher incidence of comorbidities observed in this population.