Opinion
Video
Key Clinical Considerations for the Management of SMA
Author(s):
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.
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.
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