Safety of intrathecal treatment with nusinersen therapy with spinal muscular atrophy SMA Typ 2/3
New study from University Essen shows great results
University Essen treated a total of 28 adult SMA patients (9 patients with SMA type 2 and 19 patients with SMA type 3) aged between 18–61 years with nusinersen. The mean Revised Upper Limb Module (RULM) score at baseline in SMA type 2 and SMA type 3 patients was 9.9 ± 4.6 and 29.5 ± 8.5, respectively.
The data demonstrate the feasibility and tolerability of intrathecal treatment with nusinersen in adults with SMA type 2 and type 3, but this is a logistically challenging, particularly in patients with SMA type 2 and in patients with spondylodesis.