Longitudinal data from 256 individuals with genetically confirmed SMA treated with nusinersen (Spinraza) were extracted from the SMArtCARE registry.
- Before treatment was started :
• 180 had SMA type II and were able to sit up, 107 were 5 years old or younger, 73 were older than 5 years;
• 37 had lost the ability to sit up alone;
• 39 had SMA type III and had lost the ability to walk and partially sit.
- Almost 90% had 14 months of follow-up data on nusinersen, half of them 38 months.
- Almost one third had a functionally significant improvement in their Revised Upper Limb Module (RULM) score, and almost a quarter in their Hammersmith Functional Motor Scale Expanded (HFMSE) score.
- Only 4.3% had a clinically significant deterioration in their HFMSE score and 1.2% in their RULM score.
