Opinion|Videos|February 18, 2025

The Future of Duchenne Muscular Dystrophy Management: Innovations and Emerging Strategies

Experts discuss the future of Duchenne care, emphasizing proactive management, multidisciplinary collaboration, and the need for adult-focused treatment strategies.

The management of Duchenne muscular dystrophy (DMD) is poised for significant evolution over the next five years, driven by advances in gene therapy, novel pharmacologic agents, and personalized care strategies. Emerging therapies aim not only to slow disease progression but also to address the underlying genetic causes, potentially transforming long-term outcomes. Artificial intelligence (AI) is expected to play a growing role in DMD care, enhancing diagnostic accuracy, predicting disease trajectories, optimizing treatment selection, and monitoring patient response through data-driven insights. Panelists emphasize the importance of integrating multidisciplinary care with these innovations, ensuring that advances translate into meaningful improvements in function, quality of life, and caregiver support. Future management will likely combine precision medicine, real-world evidence, and technological tools to tailor therapies to individual patient needs, while continued collaboration among clinicians, researchers, and families remains essential to maximize the benefits of emerging interventions.


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