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Articles

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 20, 2024. 

NeurologyLive® Friday 5 — December 20, 2024

Managing ALS, particularly its respiratory complications, requires a collaborative multidisciplinary team—including neurologists, pulmonologists, and respiratory therapists—working together to address the complexities of respiratory failure, the leading cause of death in these patients.

Breathing Life Into ALS Care: The Vital Role of Multidisciplinary Teams

Although the primary goal was not met, prasinezumab demonstrated encouraging effects on secondary and exploratory measures, such as time to worsening motor function, Clinical Global Impression of Change, MDS-UPDRS motor scores.

Parkinson Agent Prasinezumab Misses Primary End Point But Shows Positive Effects in Other Outcomes

Videos

The assistant professor of neurosurgery and neuroscience at Mayo Clinic Arizona discussed an ongoing early-stage study assessing the therapeutic potential of NRTX-1001 nerve cell therapy in drug-resistant unilateral mesial temporal lobe epilepsy. [WATCH TIME: 5 minutes]

Studying Nerve Cell Therapy NRTX-1001 to Treat Mesial Temporal Lobe Epilepsy: Jonathan Parker, MD, PhD

Assistant professor of neurosurgery and neuroscience at Mayo Clinic Arizona

, has been with the team since September 2022. Follow her on Twitter 

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

FDA Grants MDA Kickstart Program Rare Pediatric Disease Designation and Orphan Drug Designation in Congenital Myasthenic Syndrome

IJMSC Insights offers a closer look at the latest research and the people behind it from the community of the International Journal of Multiple Sclerosis Care (IJMSC) and the Consortium of Multiple Sclerosis Centers (CMSC).

IJMSC Insights: Blanca De Dios Perez on Employment and Multiple Sclerosis

Pediatric epilepsy specialist at University of California, San Francisco.

At AES 2024, a duo of pediatric epilepsy specialists discussed how implementation science, validated blood biomarkers, and cross-disciplinary collaboration are advancing epilepsy care and closing research gaps. [WATCH TIME: 3 minutes]

Advancing Epilepsy Research Through Implementation Science and Biomarkers: Adam Numis, MD; Laura Kirkpatrick, MD

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats.

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Treatment Advances in Targeting Underlying Pathophysiology of Myasthenia Gravis

Complement Inhibitors for Treatment of Generalized Myasthenia Gravis

Video content above is prompted by the following:

Complement inhibitor agents have been approved by the FDA for treatment of gMG. Provide an overview of their mechanism of action and your overall impressions of the efficacy and safety of these agents.

Nicholas J. Silvestri, MD, FAAN; Ali A. Habib, MD; Neelam Goyal, MD; and Katherine Ruzhansky, MD, MS, explore the multifaceted aspects of generalized myasthenia gravis, from its clinical presentation and diagnosis to emerging targeted therapies, emphasizing the importance of personalized treatment approaches, discussing recent clinical trial data, and addressing ongoing challenges in both adult and pediatric populations.

Panelists discuss how myasthenia gravis presents clinically in adults and children, factors contributing to its underdiagnosis, and symptoms that should prompt specialist consultation.

Generalized Myasthenia Gravis Presentation and Diagnosis

Panelists discuss how the classification of generalized myasthenia gravis (gMG) informs and guides treatment strategies for patients.

Generalized Myasthenia Gravis Classification and Treatment Strategy

Panelists discuss how the current understanding of myasthenia gravis pathophysiology is informing the development of targeted treatments, alongside reviewing the standard of care for generalized myasthenia gravis (gMG).

Pathophysiology and Standard of Care in Generalized Myasthenia Gravis

Panelists discuss how various outcome measures are utilized in clinical practice to assess and monitor patients with generalized myasthenia gravis.

Outcome Measures in Generalized Myasthenia Gravis

Panelists discuss how complement inhibitors work to treat generalized myasthenia gravis (gMG), evaluating their efficacy and safety profiles based on FDA approvals and clinical experience.

Panelists discuss how various approved and investigational FcRn-targeting agents differ in their mechanisms of action for treating generalized myasthenia gravis (gMG).

Neonatal Fc Receptors in Treatment of Generalized Myasthenia Gravis

Panelists discuss how specific disease characteristics and individual patient factors influence their decision-making process when selecting treatments for generalized myasthenia gravis.

Factors Guiding Treatment Selection in Generalized Myasthenia Gravis

Panelists discuss how they approach combination therapy in generalized myasthenia gravis (gMG), including patient selection and timing, while also examining how the availability of newer targeted treatments has influenced their approach to managing newly diagnosed patients.

Combination Therapy in Generalized Myasthenia Gravis and Treatment of Newly Diagnosed Patients

Panelists discuss how recent phase 3 trial results for nipocalimab and batoclimab, both neonatal Fc receptor (FcRn) inhibitors, demonstrate efficacy and safety in different generalized myasthenia gravis patient populations, comparing these findings with earlier phase 2 data and evaluating their potential impact on treatment approaches.

Nipocalimab and FcRn Inhibitors in Generalized Myasthenia Gravis

Panelists discuss how they approach and potentially adapt the dosing and administration regimens of approved and investigational FcRn inhibitors in their myasthenia gravis patients, considering the prescribed guidelines and individual patient needs.

Dosing Regimens and Treatment Efficacy of FcRn Inhibitors in Generalized Myasthenia Gravis

Panelists discuss how they select patients for FcRn and complement inhibitor therapies, integrate these treatments into long-term management plans, monitor patients on these therapies, and approach treatment for those with inadequate disease control on traditional or targeted therapies for generalized myasthenia gravis.

Use of Targeted Agents in Generalized Myasthenia Gravis

Panelists discuss how generalized myasthenia gravis (gMG) presents unique diagnostic challenges in pediatric populations, review current standard-of-care management strategies for children and adolescents, and highlight promising investigational treatments for pediatric gMG.

Management of Generalized Myasthenia Gravis in Pediatric Patients

Panelists discuss how certain aspects of myasthenia gravis management remain challenging, identifying ongoing unmet needs in diagnosis, treatment, and patient care.

Unmet Needs in Generalized Myasthenia Gravis

Panelists discuss how emerging agents for generalized myasthenia gravis (gMG) show promise in addressing current treatment gaps, highlighting specific therapies they find particularly exciting for future management of the condition.

Generalized Myasthenia Gravis Pipeline

Panelists discuss how recent data presented at the 2024 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) conference may impact clinical practice for myasthenia gravis, highlighting emerging treatments they find particularly promising for future management of the condition.

New Data from AANEM 2024 in Generalized Myasthenia Gravis

Panelists discuss how a multidisciplinary approach involving various specialists is crucial in managing generalized myasthenia gravis (gMG), offering final insights and recommendations for health care professionals treating gMG patients.

Clinical Pearls for Management of Generalized Myasthenia Gravis

Peer Exchange

Complement inhibitor agents, approved by the FDA for treating generalized myasthenia gravis (gMG), function by targeting the complement cascade, specifically inhibiting the C5 component. This action prevents the formation of the membrane attack complex, thereby reducing neuromuscular junction damage. Clinical trials have demonstrated significant efficacy in improving muscle strength and reducing disease symptoms. Safety profiles are generally favorable, with manageable adverse effects. These agents represent a promising advancement in gMG management, offering a targeted therapeutic approach that addresses the underlying pathophysiology of the disease.

 Peer Exchange video series features panels of expert physicians engaging in in-depth discussions about practice-changing treatments, challenges in care, and the future paradigm of management for neurologic diseases.

Complement Inhibitors for Treatment of Generalized Myasthenia Gravis

Key Takeaways

EP: 1.Generalized Myasthenia Gravis Presentation and Diagnosis

EP: 2.Generalized Myasthenia Gravis Classification and Treatment Strategy

EP: 3.Pathophysiology and Standard of Care in Generalized Myasthenia Gravis

EP: 4.Outcome Measures in Generalized Myasthenia Gravis

EP: 5.Complement Inhibitors for Treatment of Generalized Myasthenia Gravis

EP: 6.Neonatal Fc Receptors in Treatment of Generalized Myasthenia Gravis

EP: 7.Factors Guiding Treatment Selection in Generalized Myasthenia Gravis

EP: 8.Combination Therapy in Generalized Myasthenia Gravis and Treatment of Newly Diagnosed Patients

EP: 9.Nipocalimab and FcRn Inhibitors in Generalized Myasthenia Gravis

EP: 10.Dosing Regimens and Treatment Efficacy of FcRn Inhibitors in Generalized Myasthenia Gravis

EP: 11.Use of Targeted Agents in Generalized Myasthenia Gravis

EP: 12.Management of Generalized Myasthenia Gravis in Pediatric Patients

EP: 13.Unmet Needs in Generalized Myasthenia Gravis

EP: 14.Generalized Myasthenia Gravis Pipeline

EP: 15.New Data from AANEM 2024 in Generalized Myasthenia Gravis

EP: 16.Clinical Pearls for Management of Generalized Myasthenia Gravis