Emerging Therapies and Patient-Centered Progress in Neuromuscular Medicine: Amit Sachdev, MD, MS

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"The science is awesome, but ultimately we need to understand the patient voice. I think the FDA has taught us that it is looking for patient-reported outcomes, and that’s really where I’d like to see development in the field—understanding how to capture the patient experience."

The Myasthenia Gravis Foundation of America (MGFA) Scientific Session, held at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 29 to November 1, in San Francisco, California, brought together researchers, neurologists, early-career investigators, and other clinicians to discuss recent advances in myasthenia gravis (MG) research through concise, data-driven presentations.¹ Topics in the session included the identification and validation of biomarkers to support earlier diagnosis, predict clinical outcomes, and assess response to immunosuppressive therapies, as well as their application in clinical trials.

Additional areas of discussion included investigations into MG disease mechanisms, with an emphasis on immune dysregulation and loss of self-tolerance across the disease course, and the development of targeted therapeutic strategies designed to reduce off-target effects and optimize use of existing treatments. Presentations also addressed patient-centered outcomes, including the impact of MG on daily functioning, treatment priorities, comorbidities, adverse effects, and financial burden, as well as considerations specific to pediatric MG, such as safety, treatment strategies, and long-term outcomes.

In an interview at AANEM 2025 with CGTLive®, our sister publication, Amit Sachdev, MD, MS, associate chief medical officer at Michigan State University, reflected on the continued growth of the meeting and its expanding focus on neuroimmunology. He emphasized the impact of recent clinical trials on improving clinical understanding of MG management. He also highlighted early but promising developments in genetic muscle disease therapies alongside transformative approaches. During the conversation, Sachdev underscored the increasing importance of integrating patient-reported outcomes into clinical development.

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REFERENCES
1. MGFA Scientific Session. Presented at: 2025 AANEM; October 29 to November 1; San Francisco, California. Accessed January 23, 2026. https://myasthenia.org/wp-content/uploads/2025/09/2025-Scientific-Session-at-AANEM-Agenda_81125_FINAL.pdf