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).
Clinical Brief: Treatment Approaches for SMA in Older Patients
Main Discussion Topics
Key Points for Physicians
Notable Insights
The panel emphasized that disease phenotype is determined by inherent disease characteristics and secondary atrophy from disuse, creating a combined progression pattern that affects treatment approaches.
Clinical Significance
Understanding SMA as a life span condition requiring individualized care based on functional status and SMN2 copy numbers is essential for establishing realistic treatment expectations and goals in older patients.
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