Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

EP: 1.Overview of Gene Therapy Approaches and Impacts

EP: 2.Key Factors in Gene Vector Selection

EP: 3.Viral Vectors in Gene Therapy

EP: 4.Navigating Gene Therapy Eligibility: The Role of Genetic Testing

EP: 5.Evaluating Transformative Gene Therapies

EP: 6.Shifting the Treatment Paradigm in Spinal Muscular Atrophy

EP: 7.Developments in Muscular Dystrophy Gene Therapy

EP: 8.Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD

Now Viewing

EP: 9.Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

EP: 10.Duchenne Muscular Dystrophy Treatment Landscape Review

EP: 11.Strategic Insights Into Evolving Gene Therapy Trends

EP: 12.Sustainable Gene Therapy Coverage Approaches

EP: 13.Interpreting Clinical Data to Guide Coverage Decisions for Rare Disease Therapies

EP: 14.Coverage of Gene Therapies: Balancing Cost, Clinical Impact, and Durability of Effect

EP: 15.Innovative Payment Models for Gene Therapies

EP: 16.Ethical Implications of Patient Access Restrictions

EP: 17.Enhancing Collaboration for Patient-Centered Care in Rare Diseases

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.