- Center on Health Equity & Access
- Clinical
- Health Care Cost
- Health Care Delivery
- Insurance
- Policy
- Technology
- Value-Based Care
Final Panel Thoughts on the Management of SMA
Panelists discuss how spinal muscular atrophy (SMA) treatment will evolve over the next 5 years, likely incorporating improved gene therapy delivery systems, combination therapies, and rehabilitation models, while maintaining individualized approaches for each patient.
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
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.
