The results of the first of three parts of the DEVOTE trial evaluating higher doses of Spinraza® (nusinersen) have been published:
- this first part involved six patients with SMN1-related proximal spinal muscular atrophy (SMA), aged between 6 and 12 years, whose symptoms appeared after the age of 6 months and who were followed up for 10 months,
- it evaluated the 28 mg dose of Spinraza® (nusinersen), received at D1, D15, D29 then D149 and D269,
- It showed that the treatment was well tolerated by all participants (the side-effects were related to the lumbar puncture) and that it was effective in improving or stabilising motor function (although this was not one of the evaluation criteria for this part of the trial).
The other two parts of the trial, a double-blind trial comparing 50mg then 28mg versus 12mg, and an open-label trial testing 50mg then 28mg, are ongoing.
