Unmet Needs of Older Patients Living With SMA

EP: 1.Describing SMA Progression Over Time

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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.