Duchenne Muscular Dystrophy Treatment Landscape Review

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early intervention and comprehensive care.

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

Haumschild moderates a discussion with Pak, who provides an insightful comparison of treatment options for Duchenne muscular dystrophy (DMD). Pak highlights the potential benefits of gene therapy in preserving muscle function and slowing disease progression, contrasting it with the limitations and adverse effects of steroid-based therapies. She underscores the importance of early intervention and holistic care in managing DMD, stressing the need for vigilant monitoring and timely intervention to address complications like respiratory and cardiac issues. Additionally, Pak discusses emerging challenges in gene therapy, including distribution efficiency and high treatment costs, prompting a conversation about health care decision-making and the imperative of collaborative efforts to ensure optimal patient outcomes while navigating cost constraints. Haumschild and the panelists delve into the complexities of treatment selection, inclusion criteria, and cost containment strategies, underscoring the critical role of multidisciplinary care teams in advancing DMD management paradigms.

Video synopsis is AI-generated and reviewed by AJMC® editorial staff.

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