Key Clinical Considerations for 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

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EP: 8.Key Clinical Considerations for 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.