Tina Duong MPT, PhD, serves as a senior research scientist of neurology at Stanford Medicine.
Final Panel Thoughts on the Management of SMA
Panelists 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.
Optimizing Multidisciplinary Care Approaches for the Future of SMA Management
Panelists 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.
Insurance Considerations for SMA Therapies
Panelists 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.
Other Investigational Treatment Options for SMA
Panelists 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.
Evaluating the Potential of Intrathecal Gene Therapy
Panelists 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.
Assessing Different Factors When Selecting a Treatment
Panelists 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.
Key Clinical Considerations for the Management of SMA
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.
Splicing Modifiers and Gene Therapy for SMA
Panelists 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.
Introduction to Therapies Used to Treat SMA
Panelists 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.
The Importance of Supportive Care for Patients Living With SMA
Panelists 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.
Describing SMA Progression Over Time
Panelists 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).
Understanding SMA Subtypes and the Impact of Treatment Advances
The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term management strategies, and adequate muscle function monitoring are all important aspects of SMA care.
Redefining Functional Measures in SMA: Insights From Clinical Research
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately measure patient function across different age groups.