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Unmet Needs of Older Patients Living With SMA
Panelists discuss how the needs of older patients with spinal muscular atrophy (SMA) differ from infants, focusing on independence, education, career goals, fatigue management, and quality of life rather than just motor milestones.
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: Addressing Distinct Needs of Older Patients With SMA
Main Discussion Topics
- Differentiation of needs between pediatric and adult patients with SMA
- Importance of realistic functional goal-setting based on patient's current capabilities
- Multifaceted challenges faced by adult patients with SMA beyond motor function
- Critical life stages and stressors that can accelerate functional decline in patients with SMA
Key Points for Physicians
- Adult patients with SMA prioritize independence, education/work capabilities, fatigue management, and prevention of further decline.
- Older patients have greater autonomy in treatment decisions and face unique challenges, including reproductive concerns.
- Crisis points (puberty, growth spurts, illness/injury, aging) require heightened vigilance as they can accelerate decline.
- Treatment expectations should be individualized through provider-patient conversations about realistic goals.
Notable Insights
Providers should recognize that adult patients may not perceive subtle year-to-year changes in function but looking back over 5- to 10-year intervals reveals disease progression that requires ongoing management.
Clinical Significance
Treatment approaches for older patients with SMA must extend beyond motor function to address independence, daily living activities, education/career participation, and reproductive concerns through individualized, patient-centered care plans.
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