The study aims at understanding the efficiency and safety of using Nusinersen in subjects with SMA (spinal muscular atrophy).
The researchers collected studies on subjects above 18 who were diagnosed with either SMA2 or SMA3. They collected studies where data of baseline and six months were present.
In total, 116 subjects were selected with a mean age of 34. The HFMSE scale was higher in the subjects with SMA3 from baseline to T6 (six months). There was no correlation between the score and the subgroups. The RULM of SMA3 improved significantly from baseline to T14. The subjects with SMA2 showed no changes in HFMSE or RULM between T0 and T6 or T14. However, those subjects found some development in RULM in the residual baseline. From T6 to T14, the number of patients showing significant improvement increased from 53% to 69%.
The study proved that Nusinersen is effective and safe to use in patients with SMA2 and SMA3. The subjects with SMA3 showed progression over time. However, the effects of Nusinersen on residual motor function in subjects with advanced conditions are not clear.