Navigating new motor function trajectories: Consensus recommendations for assessment in the era of newborn screening and early treatment in SMA

Key Points

• The aim is to outline motor function outcome measures (MF-OMs) for assessing motor progress in infants with SMA.
• Achieved consensus among clinicians with expertise in SMA.
• Developed relevant MF-OMs to track motor function from infancy through childhood.
• Emphasized routine data collection using the MF-OMs for better research collaboration.
• Establishing consensus on MF-OMs facilitates tracking of motor progress.
• Routine data collection is expected to enhance understanding of disease trajectories.
• Improved cohort comparisons will aid in future SMA research.

Structured PICO

Abstract