Clinical Brief: Treatment Approaches for SMA in Older Patients
Main Discussion Topics
- Introduction to SMA and the expert panel of specialists from neurology, pediatrics, and physical therapy
- Discussion of SMA progression over time, particularly focusing on functional decline in motor neurons and muscle function
- Evolution of SMA classification from traditional types (1 to 4) to more functional categories (nonsitters, sitters, walkers) due to treatment advancements
- The importance of SMN2 copy numbers in determining disease severity and potential functional outcomes
Key Points for Physicians
- SMA is now considered a life span disease with an extended adult phase, especially with newer interventions.
- For older patients, treatment goals focus on slowing functional decline rather than achieving developmental milestones.
- SMN2 copy numbers remain important predictors of maximum function, even with disease-modifying therapies.
- Classification has evolved from types 1 to 4 to functional categories (nonsitters, sitters, walkers) that better reflect treatment outcomes.
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.
