Safety of intrathecal treatment with nusinersen therapy with SMA

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.

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Liz Andersson

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2 Responses to Safety of intrathecal treatment with nusinersen therapy with SMA

  1. MiaMia says:

    👍 Interesting study, this showing that is it a challenge to administer spinraza via lumbar puncture in adults with SMA and scoliosis

    Even if they having an surgery to scoliosis

  2. Absolutely and agree, there are some patients in Germany, who have problems that they have no liquid. Even though use a port as access, there is no guarantee that you can give Spinraza always.

    As known from the treatment of cancer, ports can also close after a short time.. Of course, by an existing scoliosis which often goes to the spine, it is even more problematic..