Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

Jessica Nance, MD, MS, discusses the significance of surrogate end points, like functional assessments, in clinical trials of treatments for Duchenne muscular dystrophy and their implications for clinical benefits.

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 engages Nance in a comprehensive discussion on the importance of surrogate end points, particularly functional assessments, in Duchenne muscular dystrophy (DMD) trials. Nance elucidates the challenges of interpreting changes in functional scores and highlights the nuanced implications for treatment evaluation. She emphasizes the substantial impact even slight changes in functional abilities can have on patients’ lives, underscoring the complexity of assessing clinical benefit in DMD. Moreover, Nance provides valuable insights into the potential expansion of eligibility criteria for gene therapies like delandistrogene moxeparvovec-rokl, considering safety concerns and efficacy expectations across different age groups. She advocates for a broader target patient population to maximize therapeutic benefits while acknowledging the need for careful evaluation of safety profiles, especially in older patients. Nance’s expertise sheds light on the intricate balance between treatment efficacy, safety considerations, and patient outcomes in advancing DMD therapies.

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

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