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Key Clinical Considerations for the Management of SMA
Panelists discuss how disease progression despite treatment in older patients with spinal muscular atrophy (SMA) may be due to natural aging effects combined with SMA, not necessarily treatment failure, highlighting the importance of exercise, nutrition, and management of contractures.
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: Managing Progression in Patients With Treated SMA
Main Discussion Topics
- Potential continued progression despite treatment in older patients with SMA
- Effect of normal aging processes on SMA progression
- Importance of complementary therapies for optimizing outcomes
- Role of adherence in treatment efficacy and challenges with monitoring
Key Points for Physicians
- Disease progression despite treatment does not necessarily indicate treatment failure.
- Normal aging processes can compound SMA-related weakness.
- Contracture management and exercise are key complementary approaches.
- Poor treatment adherence may contribute to progression but is difficult to measure due to the lack of biomarkers.
Notable Insights
The panel emphasized that stabilization should be considered a positive outcome in older patients, especially when considering the combined effects of SMA and normal age-related decline in muscle function.
Clinical Significance
A comprehensive approach combining disease-modifying therapies with exercise, contracture management, nutrition support, and respiratory care is essential for managing progression in older patients with SMA, with realistic expectations about treatment outcomes.
Managed Care Reflections: A Q&A With A. Mark Fendrick, MD, and Michael E. Chernew, PhD
December 2nd 2025To mark the 30th anniversary of The American Journal of Managed Care (AJMC), each issue in 2025 includes a special feature: reflections from a thought leader on what has changed—and what has not—over the past 3 decades and what’s next for managed care. The December issue features a conversation with AJMC Co–Editors in Chief A. Mark Fendrick, MD, director of the Center for Value-Based Insurance Design and a professor at the University of Michigan in Ann Arbor; and Michael E. Chernew, PhD, the Leonard D. Schaeffer Professor of Health Care Policy and the director of the Healthcare Markets and Regulation Lab at Harvard Medical School in Boston, Massachusetts.
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