An Italian real-life study conducted over 3 months in nine children with type I proximal spinal muscular atrophy (SMA) (1.7-48 months) on Zolgensma®, seven of them having received Spinraza® regularly, showed that :
- participants’ motor performance improved during the three months of follow-up,
- this improvement under Zolgensma® was not as great as the initial improvement induced by Spinraza®, which could be explained by the fact that Spinraza® allowed for progress whose kinetics cannot be maintained at the same rate given the progress made,
- on the other hand, the two patients who had responded poorly to Spinraza® responded very significantly to Zolgensma,
- the age of the patient seems to be a factor, with a greater improvement in the younger patients,
- Zolgensma® was well tolerated.
However, these first real-life data from a double treatment will have to be confirmed on a larger scale and in the longer term.
