Final Panel Thoughts on the Management of SMA

EP: 1.Describing SMA Progression Over Time

EP: 2.Unmet Needs of Older Patients Living With SMA

EP: 3.Examining the Importance of Early Treatment for SMA

EP: 4.Key Functional Milestones Affected by Disease Progression

EP: 5.The Importance of Supportive Care for Patients Living With SMA

EP: 6.Introduction to Therapies Used to Treat SMA

EP: 7.Splicing Modifiers and Gene Therapy for SMA

EP: 8.Key Clinical Considerations for the Management of SMA

EP: 9.Assessing Different Factors When Selecting a Treatment

EP: 10.Evaluating the Potential of Intrathecal Gene Therapy

EP: 11.Other Investigational Treatment Options for SMA

EP: 12.Insurance Considerations for SMA Therapies

EP: 13.Optimizing Multidisciplinary Care Approaches for the Future of SMA Management

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EP: 14.Final Panel Thoughts on the Management of SMA

Clinical Brief: Future Directions in SMA Management

Main Discussion Topics

• Anticipated improvements in gene therapy delivery and safety
• Shift toward combination therapy approaches
• Evolution from palliative to rehabilitation-focused care models
• Need for ongoing basic science research to address emerging clinical questions

Key Points for Physicians

• Future gene therapies may utilize improved viral capsids with reduced liver targeting to minimize adverse events.
• Combination therapies targeting different aspects of SMA pathophysiology will likely become the standard of care.
• The care model is evolving to parallel approaches used in spinal cord injury and traumatic brain injury.
• Translational research between basic scientists and clinicians remains crucial to address emerging questions.

Notable Insights

The panel emphasized that while current treatments have transformed SMA from a frequently fatal disease to a manageable chronic condition, they are not curative and require continued research for optimization.

Clinical Significance

The future of SMA management will likely involve individualized combination approaches with safer gene delivery systems, targeted muscle-enhancing therapies, and comprehensive rehabilitation strategies to maximize functional outcomes throughout patients’ life span.