Examining the Importance of Early Treatment for SMA

EP: 1.Describing SMA Progression Over Time

EP: 2.Unmet Needs of Older Patients Living With SMA

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EP: 3.Examining the Importance of Early Treatment for SMA

EP: 4.Key Functional Milestones Affected by Disease Progression

Clinical Brief: Treatment Timing for Patients With SMA Across Age Groups

Main Discussion Topics

• Critical importance of early intervention in patients with newly diagnosed SMA
• Differences in treatment urgency between infants and adults with SMA
• Challenges in measuring treatment response in adult patients
• Limitations of standard assessment tools for different functional levels

Key Points for Physicians

• Infant SMA treatment is a medical emergency; progression can occur within days.
• While not requiring 24 to 48 hour intervention, adult SMA treatment remains urgent to preserve remaining motor neurons.
• Motor neurons cannot regenerate once lost, making preservation of existing neurons critical.
• Stabilization may be considered a treatment success, especially in adult patients.

Notable Insights

Assessment tools developed for specific age groups or functional levels may have floor/ceiling effects that limit their utility in other populations, necessitating alternative evaluation methods to document meaningful changes.

Clinical Significance

While treatment timing is critical across all SMA age groups, physicians must adapt intervention timelines and outcome measurements to appropriately assess and document treatment benefits in older patients.