Assessing Different Factors When Selecting a Treatment

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

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EP: 9.Assessing Different Factors When Selecting a Treatment

EP: 10.Evaluating the Potential of Intrathecal Gene Therapy

Clinical Brief: Comorbidities and Changing Phenotypes in Patients With Treated SMA

Main Discussion Topics

• Impact of comorbidities on treatment decisions and management
• Challenges in addressing orthopedic issues in functionally improving patients
• Evolving patterns of scoliosis and spinal deformities in treated patients
• Reproductive considerations for patients with SMA

Key Points for Physicians

• Treatment has changed the natural history of SMA, creating new clinical challenges in managing comorbidities.
• Orthopedic interventions (for dislocated hips, scoliosis) require reassessment as patients gain function.
• Kyphosis is becoming more prominent than typical scoliosis in treated patients.
• Genetic counseling remains essential as disease-modifying therapies do not alter genetic status.

Notable Insights

The panel noted that disease-modifying treatments are not curative and do not change genetic inheritance patterns, necessitating continued genetic counseling for family planning.

Clinical Significance

As disease-modifying therapies alter the natural history of SMA, physicians must reconsider traditional approaches to comorbidities and develop new management paradigms for this evolving clinical landscape.