In untreated patients with SMA, motor function tended to improve in younger patients before beginning a period of decline from ages 5 to 13 years old¹

Retrospective study data* showing age-related motor function in patients with SMA type 2¹

Adapted with permission from Mercuri et al., 2019.

*Results from a single-center, retrospective study involving 73 patients in total, 28 of whom had more consistent longitudinal data and were assessed before the age of 5 years and had been followed up for ~5 years or longer. Patients enrolled in clinical trials were included until the baseline of the trial.¹

Progressive loss of motor function with spinal muscular atrophy (SMA) was nonlinear¹

•   Patients with SMA type 2 appeared stable over short intervals and children under 5 years old showed an overall trend to improve motor function
•  Steep decline typically occurs between ages 5 and 13, with relative stabilization after age 13
 
Understanding the trajectory of SMA can help improve outcomes¹

•  Baseline Hammersmith Functional Motor Scale — Expanded (HFMSE) score predicted the rate and magnitude of decline
•  Patients with lower baseline scores experience earlier onset of scoliosis and a higher need for noninvasive ventilation
 
The annual loss in HFMSE score between the ages of 5 and 13 years old was about 2 points per year—each point lost may be considered clinically meaningful by patients and caregivers.^1,2

HFMSE, Hammersmith Functional Motor Scale — Expanded; SMA, spinal muscular atrophy.