Julie A. Parsons, MD; John Brandsema, MD; and Tina Duong, MPT, PhD, discuss how management approaches for older patients with spinal muscular atrophy (SMA) differ from those for infants, highlighting the importance of individualized treatment goals focused on independence, fatigue management, quality of life, and the challenges of measuring outcomes with currently available assessment tools, while also exploring current and emerging therapies, including splice modifiers and potential intrathecal gene therapy options.
EP. 1: Describing SMA Progression Over Time
May 15th 2025Panelists discuss how spinal muscular atrophy (SMA) progresses over time, affecting motor function differently in older patients vs infants, with experts noting the shift from traditional classification (types 1 to 4) to functional categories (nonsitters, sitters, walkers).
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EP. 2: Unmet Needs of Older Patients Living With SMA
May 15th 2025Panelists 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.
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EP. 5: The Importance of Supportive Care for Patients Living With SMA
May 29th 2025Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory care, psychosocial support, and multidisciplinary approaches to help patients achieve independence and improved quality of life.
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EP. 6: Introduction to Therapies Used to Treat SMA
May 29th 2025Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers (nusinersen [Spinraza] and risdiplam [Evrysdi]) for older patients.
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EP. 7: Splicing Modifiers and Gene Therapy for SMA
June 5th 2025Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered intrathecally quarterly, both showing similar safety and efficacy profiles.
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EP. 8: Key Clinical Considerations for the Management of SMA
June 5th 2025Panelists 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.
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EP. 9: Assessing Different Factors When Selecting a Treatment
June 12th 2025Panelists discuss how comorbidities such as scoliosis, dislocated hips, and nutritional issues affect spinal muscular atrophy (SMA) treatment decisions, with treatment approaches evolving as patients' functional abilities improve with disease-modifying therapies.
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EP. 10: Evaluating the Potential of Intrathecal Gene Therapy
June 12th 2025Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest efficacy with fewer systemic adverse effects than intravenous administration, though safety concerns and limited data remain.
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EP. 11: Other Investigational Treatment Options for SMA
June 19th 2025Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, hold promise for addressing fatigue and improving muscle function in older patients.
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EP. 12: Insurance Considerations for SMA Therapies
June 19th 2025Panelists discuss how insurance challenges impact spinal muscular atrophy (SMA) care, with prior authorizations becoming more streamlined over time but limitations on physical therapy sessions and inequitable access to treatments remain significant barriers.
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EP. 13: Optimizing Multidisciplinary Care Approaches for the Future of SMA Management
June 26th 2025Panelists discuss how communication between clinicians, patients, and payers could improve equitable access to spinal muscular atrophy (SMA) treatments, while acknowledging the complex value assessment of high-cost therapies vs improved quality and length of life.
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EP. 14: Final Panel Thoughts on the Management of SMA
June 26th 2025Panelists discuss how spinal muscular atrophy (SMA) treatment will evolve over the next 5 years, likely incorporating improved gene therapy delivery systems, combination therapies, and rehabilitation models, while maintaining individualized approaches for each patient.
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